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  • Fabry Disease: Patients' Real-World Problems and How to Manage Them

    Fabry Disease: Patients' Real-World Problems and How to Manage Them
    2026/09/09
    Living with Fabry disease can involve much more than managing the underlying disease. Patients may experience chronic pain, fatigue, headaches, cognitive concerns, hearing difficulties, and complications involving the heart and kidneys. Coordinating care among multiple specialists can add another layer of complexity.

    In this episode, Staci Kallish, DO, Associate Professor of Clinical Medicine at the Perelman School of Medicine at the University of Pennsylvania, and Dawn Laney, MS, CGC, CCRC, Genetic Counselor and Program Leader of the Lysosomal Storage Disease Center at Emory University School of Medicine, discuss the real-world challenges faced by people living with Fabry disease.

    The conversation features candid patient perspectives on four key areas of Fabry care:
    • Managing symptoms: Why neuropathic pain, abdominal pain, headaches, fatigue, and other persistent symptoms may require approaches beyond Fabry-specific therapy.
    • Managing cardiac and stroke risk: Why routine tests may not always provide the full picture and how experienced Fabry specialists can work with local cardiologists and other providers.
    • Protecting kidney health: The importance of distinguishing Fabry-related kidney disease from other kidney problems and ensuring that nephrologists understand the unique risks associated with Fabry disease.
    • Coordinating a multidisciplinary care team: How a “quarterback” or medical home can help connect geneticists, cardiologists, nephrologists, primary care physicians, and other specialists.

    Drs. Kallish and Laney also discuss the importance of recognizing invisible symptoms, understanding individual pain triggers, using recommended schedules of assessments, accessing therapies closer to home, and taking advantage of patient advocacy and financial assistance resources.

    For patients and families living with Fabry disease, the message is clear: effective care requires more than treating the disease—it requires understanding the person living with it.

    This educational program is a non-CME activity made possible by an unrestricted grant from Amicus Therapeutics.

    Produced by CheckRare.

    Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert interviews, and weekly news.

    Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.
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    38 分
  • Beyond AChR: Recognizing and Treating MuSK Ab+, LRP4, Ab+ and Triple Seronegative Myasthenia Gravis

    Beyond AChR: Recognizing and Treating MuSK Ab+, LRP4, Ab+ and Triple Seronegative Myasthenia Gravis
    2026/09/01
    This program is supported by educational grants from argenx. To obtain CME credit, visit https://checkrare.com/learning/p-beyond-achr-recognizing-and-treating-musk-ab-lrp4-ab-and-triple-seronegative-myasthenia-gravis/This accredited CME program examines best practices for diagnosing and treating patients with ultra-rare forms of myasthenia gravis. The most prevalent form of myasthenia gravis is associated with antibodies against the acetylcholine receptor (AChR Ab+). However, 15- 20% of patients with myasthenia gravis are not AChR Ab+. These clinically important and underrecognized subsets of patients have antibodies against muscle-specific kinase (MuSK Ab+), lipoprotein receptor-related protein 4 (LRP4 Ab+), or test negative for all known antibodies (triple seronegative MG). These patients present unique diagnostic and treatment challenges that necessitate the need for experts to provide guidance that can improve how these patients are better managed.Led by Drs. Neelam Goyal and Christyn Edmundson, this activity used case studies involving three different patients suspected of having MuSK Ab+, LRP4, Ab+ and Triple Seronegative Myasthenia Gravis to discuss how they assess those patients, diagnose them properly, and decide which treatment option is most appropriate for that patient. Target AudienceThis activity has been designed to meet the educational needs of physicians specializing in neurology, ophthalmology, rheumatology, and family practice. Other members of the care team may also participate.Learning ObjectivesAfter participating in the activity, learners should be better able to:Describe the current hypotheses regarding disease mechanisms in MuSK Ab+, LRP4 Ab+, and triple seronegative MG and apply this mechanistic understanding to diagnostic reasoning and treatment planning.Recognize the subtype-specific clinical features of MuSK Ab+, LRP4 Ab+, and triple seronegative MG and apply an appropriate diagnostic workupIdentify patients with MuSK Ab+, LRP4 Ab+, or triple seronegative MG who are suboptimally controlled on traditional therapies and apply subtype-specific evidence to guide timely transition to newer therapies.Articulate the distinct treatment burden and disease progression patterns in MuSK+, LRP4+, and triple seronegative MG and apply treatment goals that reflect the potential benefits of targeted over traditional therapy.Neelam Goyal, MDClinical Professor, Neuromuscular MedicineStanford UniversityStanford, CAChristyn Edmundson, MDNeurologistSwedish Neuroscience Institute Seattle, WADisclosure StatementAccording to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.Disclosure of relevant financial relationships are as follows:Faculty Educator/PlannerDr. Goyal discloses the following relevant financial relationships with ineligible companies:Advisory Board/Consultant: argenx, Alexion, UCB, Janssen, Amgen, Novartis, Immunovant*, Dianthus, Seismic*, Cartesian*, EMD Serono* (*Relationship has ended)Research Grant: argenxDr. Edmunson discloses the following relevant financial relationships with ineligible companies:Advisory Board/Consultant: Alnylam, Alexion, Amgen, argenx, Kyverna, UCBSpeaker’s Bureau: Alnylam, Alexion, Amgen, argenx, UCBAcademy and CheckRare CE planners and reviewers for this activity have no relevant financial relationships with any ineligible companies.This activity will review off-label or investigational information.The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.Accreditation and Credit DesignationIn support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.PhysiciansAmerican Academy of CME, Inc., designates this enduring material for a maximum of 0.75 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. ...
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    45 分
  • Myasthenia Gravis Clinical Research Highlights

    Myasthenia Gravis Clinical Research Highlights
    2026/08/02
    This program is supported by educational grants from argenx and UCB, Inc.This accredited CME program highlights the latest clinical research about myasthenia gravis, a rare, autoimmune disease that targets the neuromuscular junction. To obtain CME credit for this activity, visit https://checkrare.com/learning/p-myasthenia-gravis-clinical-research-highlights-2026/Management of myasthenia gravis is highly individualized and depends largely on the disease subtype and comorbidities of each patient. Recent clinical studies have expanded the evidence base for approved and emerging therapies, providing clinicians with new information that may inform treatment selection and long-term disease management. Highlights from the American Academy of Neurology Annual Meeting (AAN 2026) include data on approved therapies, their use in specific patient populations, and investigational approaches currently under evaluation.Led by Drs. Ratna Bhavaraju-Sanka and Chilvana Patel, this program summarizes clinically relevant findings and discusses their potential implications for enhancing the care of patients with myasthenia gravis.Target AudienceThis activity has been designed to meet the educational needs of physicians specializing in neurology, ophthalmology, rheumatology, and family practice. Other members of the care team may also participate.Learning ObjectivesAfter participating in the activity, learners should be better able to:Summarize key advances in MG research presented at AAN 2026 and discuss their potential implications for diagnosis, monitoring, and long-term management.Communicate clinically relevant updates in MG research to members of the multidisciplinary care team to support coordinated, evidence-based patient care.Ratna Bhavaraju-Sanka, MD, FAANAssociate Professor,University of Texas Health, San Antonio, TXChilvana Patel, MD, FANAClinical Associate Professor,University of Texas Medical Branch, Galveston, TXDisclosure StatementAccording to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.Disclosure of relevant financial relationships are as follows:Faculty Educator/PlannerDr. Bhavaraju-Sanka discloses the following relevant financial relationships with ineligible companies:Consultant: Amgen, argenxDr. Patel has no relevant financial relationships with any ineligible companies.Academy and CheckRare CE planners and reviewers for this activity have no relevant financial relationships with any ineligible companies.This activity will review off-label or investigational information.The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.Accreditation and Credit DesignationIn support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.PhysiciansAmerican Academy of CME, Inc., designates this enduring material for a maximum of 0.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPsOther members of the care team will receive a certificate of participation.There are no fees to participate in the activity. Participants must review the activity information, including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre- and post-program assessments. Your certificate will be emailed to you within 30 days.PrivacyFor more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm For more information about CheckRare’s privacy policy, please access https://checkrare.com/privacy/ContactFor any questions, please contact: CEServices@academycme.orgCopyright© 2026. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy ...
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    35 分
  • Current Issues in Gene Therapies for Lysosomal Disorders

    Current Issues in Gene Therapies for Lysosomal Disorders
    2025/12/31
    Shunji Tomatsu, MD, PhD, Professor and Head, Nemours Children’s Health, Delaware, USA; Alessandra d’Azzo, PhD, Emerita Faculty, Genetics, St. Jude Children’s Research Hospital, Tennessee, USA; Merve Emecen Sanli, MD, Associate Professor, Department of Pediatrics, University of Texas Southwestern Medical Center, Texas, USA; and Ryan Colburn, patient with Pompe disease and president of Odimm Inc, discuss new and emerging gene therapies for lysosomal disorders.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees.To obtain CME/CE credit, please visit https://checkrare.com/learning/p-grids2025-session6-current-issues-in-gene-therapies-for-lysosomal-disorders/ Learning ObjectivesDescribe current and emerging gene therapy data in lysosomal disorders and its clinical relevanceDescribe role of patients in gene therapy developmentFacultyShunji Tomatsu, MD, PhD, Professor and Head, Nemours Children’s HealthAlessandra d’Azzo, PhD, Emerita Faculty, Genetics, St. Jude Children’s Research HospitalMerve Emecen Sanli, MD, Associate Professor, Department of Pediatrics, University of Texas Southwestern Medical CenterRyan Colburn. Odimm, Inc.DisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Shunji Tomatsu, MD, PhD Dr. Tomatsu has received the following grants: Morquio Foundations and families: Scarlett Grifith, Bennett, A Cure for Roberts, and Morquio Conference; MPS Societies: Japanese, National, and Austrian; NIH grants: 1-R01-HD102545, NIH, NICHD, Tomatsu (PI), 1R01HD104814-01A1, NIH, NICHD, Langan, T.J. (PI), Role: Site-PI, R43HD114328-01, NIH, ACOSTA, WALTER (PI), Role: site PI, 1R43AR084638-01, NIH, MOUNZIH, KHALID (PI); Foundation of NIH: FNIH RFP NUMBER: 2022-BGTC-005 Tomatsu (PI). Alessandra d’Azzo, PhDDr. D’Azzo has no relevant financial relationships to disclose.Merve Emecen Sanli, MDDr. Sanli has no relevant financial relationships to disclose.Ryan ColburnMr. Colburn has an advisory, consulting and/or project based relationship or stock holding with: Abeona Therapeutics, Amicus Therapeutics, Astellas Gene Therapies, Avidity Biosciences, Bayer, Catalyst Pharmaceuticals, Denali Therapeutics, M6P Therapeutics, Sangamo Therapeutics, Sanofi, Solid Biosciences.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Accreditation and Credit DesignationPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesAffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC). This activity provides a maximum of 1 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic CounselorsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Genetic Counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education ...
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    1 時間 47 分
  • Theranostics and Lysosomal Disorders

    Theranostics and Lysosomal Disorders
    2025/12/31
    Duarte C. Barral, PhD, Associate Professor, NOVA Medical School, NOVA University of Lisbon, Portugal; Nuno Raimundo, PhD, Associate Professor, Department of Cellular and Molecular Physiology; Penn State College of Medicine, Pennsylvania, USA; Betul Celik, PhD, Postdoctoral Fellow, Nemours Children’s Health, Delaware, USA; and Gregory Newby, PhD, Assistant Professor, Department of Genetic Medicine, Johns Hopkins School of Medicine, Maryland, USA,discuss the principles of theranostics and its application in lysosomal disorders.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees.To obtain CME/CE credit, please visit https://checkrare.com/learning/p-grids2025-session5-theranostics-and-lysosomal-disorders/ Learning ObjectivesDescribe lysosomal and inter-organelle mechanisms that contribute to pathology in lysosomal disorders, and how these pathways are being leveraged for diagnostic and therapeutic applications.Describe current and emerging theranostic strategies for lysosomal disorders.FacultyDuarte C. Barral, PhD, Associate Professor, NOVA Medical School, NOVA University of Lisbon, Nuno Raimundo, PhD, Associate Professor, Department of Cellular and Molecular Physiology; Penn State College of MedicineBetul Celik, PhD, Postdoctoral Fellow, Nemours Children’s HealthGregory Newby, PhD, Assistant Professor, Department of Genetic Medicine, Johns Hopkins School of MedicineDisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Duarte C. Barral, PhD Dr. Barral’s group receives grant support from Sea4Us.Nuno Raimundo, PhDDr. Raimundo has no relevant financial relationships to disclose.Betul Celik, PhDDr. Celik has no relevant financial relationships to disclose.Gregory Newby, PhDDr. Newby has no relevant financial relationships to disclose.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Accreditation and Credit DesignationPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesAffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC). This activity provides a maximum of 1 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic CounselorsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Genetic Counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity. Participation CostsThere is no cost to participate in this activity.CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.comSend ...
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    1 時間 46 分
  • Expanded Applications of AI in Lysosomal Disorders

    Expanded Applications of AI in Lysosomal Disorders
    2025/12/31
    Oral Alpan, MD, Immunologist, Amerimmune, Virginia, USA; Svenja Keller, PhD student, University of Zurich, Switzerland; Shoshana Revel-Vilk, MD, PhD, Director, Gaucher Unit & Pediatric Hematology/Oncology Unit, Shaare Zedek Medical Center, Jerusalem, Israel; Patrick Deegan, MD, Consultant Metabolic Physician, University of Cambridge, UK; and Ravi Kamath, MD, PhD, Head of Musculoskeletal Radiology, Inova Health System, Virginia, USA, discuss the applications of AI in the diagnosis and treatment of lysosomal disorders.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees.To obtain CME/CE credit, visit https://checkrare.com/learning/p-grids2025-session4-expanded-applications-of-ai-in-lysosomal-disorders/Learning ObjectivesDescribe how emerging AI and machine learning technologies are advancing disease modeling and biomarker development.Describe how emerging AI and machine learning technologies are advancing therapeutic target identification across lysosomal disorders.FacultyOral Alpan, MD, Immunologist, AmerimmuneSvenja Keller, PhD student, University of ZurichShoshana Revel-Vilk, MD, PhD, Director, Gaucher Unit & Pediatric Hematology/Oncology Unit, Shaare Zedek Medical CenterPatrick Deegan, MD, Consultant Metabolic Physician, University of CambridgeRavi Kamath, MD, PhD, Head of Musculoskeletal Radiology, Inova Health SystemDisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Oral Alpan, MD Dr. Alpan has no relevant financial relationships to disclose.Svenja KellerMs. Keller has no relevant financial relationships to disclose.Shoshana Revel-Vilk, MD, PhDDr. Revel-Vilk receives grant/research support from Sanofi and Takeda. She is a member of the Speakers Bureau for Sanofi and Takeda, and a member of the Advisory Board for Takeda.Patrick Deegan, MDDr. Deegan is a consultant and advisory board member with Sanofi, Takeda, and Amicus.He also receives research support from Sanofi and Amicus.Ravi Kamath, MD, PhDDr. Kamath is on an advisory board for Intrinsic Therapeutics. He is also a consultant forSanofi, Takeda, and Spur Therapeutics.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Accreditation and Credit DesignationPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesAffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC). This activity provides a maximum of 1 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic CounselorsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Genetic Counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for ...
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    2 時間 1 分
  • Organoids and Lab-Grown Models in Lysosomal Disorders

    Organoids and Lab-Grown Models in Lysosomal Disorders
    2025/12/31
    Mia Horowitz, PhD, Tel Aviv University; Aitor Aguirre, PhD, Michigan State University, Michigan, USA; and Ying Sun, PhD, University of Cincinnati, discuss the use of organoid models in lysosomal disorder research and drug development.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees.To obtain CME/CE credit, visit https://checkrare.com/learning/p-grids2025-session3-organoids-and-lab-grown-models-in-lysosomal-disorders/Learning ObjectivesDescribe the use of heart organoid models to better understand the pathophysiology of lysosomal disorders and its clinical relevanceDescribe the use and application of brain organoid models in neuropathic Gaucher disease research and treatmentFacultyMia Horowitz, PhD, Shmunis School of Biomedicine and Cancer Research, George S. Wise Faculty of Life Sciences, Tel Aviv University.Aitor Aguirre, PhD, Associate Professor of Biomedical Engineering, Institute for Quantitative Health Science and Engineering, Chief, Division of Developmental and Stem Cell Biology (IQ), Director, MSU Stem Cell Core, Michigan State University.Ying Sun, PhD, Professor, Cincinnati Children’s Hospital Medical Center, University of Cincinnati.DisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Mia Horowitz, PhDDr. Horowitz has no relevant financial relationships to disclose.Aitor Aguirre, PhDDr. Aguirre has no relevant financial relationships to disclose.Ying Sun, PhDDr. Sun receives research support from Enkefalos Biosciences and Yuhan Corporation.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Accreditation and Credit DesignationPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesAffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC). This activity provides a maximum of 1 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic CounselorsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Genetic Counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity. Participation CostsThere is no cost to participate in this activity.CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.comSend customer support requests to cds_support+ldrtc@affinityced.comProduced by CheckRare. Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert ...
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    1 時間 36 分
  • Nanotechnology and Lysosomal Disorders

    Nanotechnology and Lysosomal Disorders
    2025/12/31
    Stephan Stern, PhD, DABT, Director of Research and Development, Nanotechnology Characterization Lab (NCL), Frederick National Laboratory for Cancer Research, Maryland, USA; and Ruben Boado, PhD, Professor of Medicine, University of California at Los Angeles, California, USA, discuss the use of nanotechnology in the treatment of lysosomal disorders.This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees.To obtain CME/CE credit, visit https://checkrare.com/learning/p-grids2025-session2-nanotechnology-and-lysosomal-disorders/Learning ObjectivesDescribe recent advances in the use of nanotechnology to treat lysosomal disordersDescribe the role of nanotechnology in addressing unmet needs in lysosomal disordersFacultyStephan Stern, PhD, DABTDirector of Research and Development, Nanotechnology Characterization Lab (NCL), Frederick National Laboratory for Cancer ResearchRuben Boado, PhDProfessor of Medicine, University of California at Los AngelesDisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Stephan Stern, PhD, DABTDr. Stern has no relevant financial relationships to disclose. Ruben Boado, PhDDr. Boado has no relevant financial relationships to disclose.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity. Conflicts of interest for presenting faculty with relevant financial interests were resolved through peer review of content by a non-conflicted reviewer.Accreditation and Credit DesignationPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Physician AssistantsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physician Assistants should claim only the credit commensurate with the extent of their participation in the activity.NursesAffinityCE is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC). This activity provides a maximum of 1 hours of continuing nursing education credit.Nurse PractitionersAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Nurse practitioners should claim only the credit commensurate with the extent of their participation in the activity.Genetic CounselorsAffinityCE designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Genetic Counselors should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other health care professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity. Participation CostsThere is no cost to participate in this activity.CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.comSend customer support requests to cds_support+ldrtc@affinityced.comProduced by CheckRare. Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert interviews, and weekly news.Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.
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