Janet Legare, MD, Professor of Pediatrics in the Division of Genetics at the University of Wisconsin, discusses week 52 results of TransCon CNP (navepegritide) in children with achondroplasia.

Achondroplasia is a genetic disorder that prevents the changing of cartilage to bone. It is characterized by dwarfism, limited range of motion at the elbows, large head size, small fingers, and normal intelligence. Achondroplasia can cause health complications such as interruption of breathing, obesity, recurrent ear infections, and an exaggerated inward curve of the lumbar spine. More serious problems include a narrowing of the spinal canal that can pinch the upper part of the spinal cord and hydrocephalus. Some people with achondroplasia may have delayed motor development early on, but cognition is normal. Achondroplasia is caused by genetic changes in the FGFR3 gene.

Navepegritide is an investigational prodrug of C-type natriuretic peptide (CNP) designed for continuous inhibition of the overactive FGFR3 pathway. Week 52 results from the randomized, double-blind, placebo-controlled ApproaCH clinical trial of navepegritide in children with achondroplasion was recently published in JAMA Pediatrics. The trial included 84 children with achondroplasia ages 2 to 11 years, randomized to navepegritide or placebo.

Treatment with navepegritide was observed to increase annualized growth velocity (AGV) at week 52 compared to placebo. Improvements were also seen in lower-limb alignment and body proportionality, with decreased upper-to-lower body segment ratio from baseline to week 52 and improved tibial-femoral angle, mechanical axis deviation, and fibula-to-tibia length ratio from baseline to week 52 compared to placebo.

Additionally, navepegritide led to numerical improvements in Achondroplasia Child Experience Measure health-related quality of life measures compared to placebo. These benefits were achieved without accelerating bone age or negatively impacting spinal curvature.

Safety and tolerability of navepegritide were similar with placebo, with the majority of adverse events being mild or moderate. Injection site reactions were low and no symptomatic hypotension or bone fractures were observed.

Navepegritide has a Prescription Drug User Fee Act (PDUFA) Date of February 28, 2026.

Chapters:
Introduction 00:00
Achondroplasia Overview 00:30
Current Management 1:22
TransCon CNP Overview 2:18
ApproaCH Clinical Trial 3:03
Next Steps 4:38
Take Home Message 5:19

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David Kuter, MD, DPhil, Professor of Medicine at Harvard Medical School, Massachusetts General Hospital, discusses treatment with rilzabrutinib in patients with immune thrombocytopenia (ITP).

ITP is an autoimmune bleeding disorder characterized by too few platelets in the blood. Symptoms may include bruising, nosebleeds or bleeding in the mouth, bleeding into the skin, and abnormally heavy menstruation.

Recently, new data from the phase 3 LUNA clinical trial (NCT04562766) was presented at the 2025 American Society of Hematology Annual Meeting (ASH 2025). Rilzabrutinib is an oral, reversible, covalent, selective Bruton tyrosine kinase inhibitor that acts through multi-immune modulation.

As of April 1, 2025, 69 of 202 patients had entered the long term extension and 57 were ongoing in the long term extension. A total of 43 patients had completed 12 months or more of follow-up in the long term extension. Median duration of ITP was 9.6 years, median number of unique prior therapies was 4, and 97% of patients had received corticosteroids (CS). Median baseline platelet count was 18×10 9 /L. Median platelet count at long term extension entry was 113×10 9 /L and median counts at long term extension follow-up visits ranged from 83-168×10 9 /L through 33 months of extension.

During the first 12 months of the extension, patients had platelet counts 50×10^9 /L or greater or between 30-50×10^9 /L and at least doubled from baseline for an average of 88% of visits. Of patients who received rilzabrutinib monotherapy or rilzabrutinib and concomitant CS and/or thrombopoietin-receptor agonists, median platelet counts at extension entry were 78×10^9 /L and 115×10^9 /L, respectively, and ranged from 78-270×10^9 /L and 90- 160×10^9 /L, respectively, through 33 months.

In the extension, 20% of patients received rescue medication. Among 49% of patients who entered the extension on concomitant CS, 29% discontinued CS use, 6% reduced their CS 50% or greater from baseline, and 18% reduced their CS dose to less than 5 mg. Median platelet counts before and after CS discontinuation at last available visit were 148×10^9 /L and 178×10^9 /L, respectively.

Improvements in fatigue scores and bleeding symptoms were generally maintained during the extension. 19% of patients had treatment-emergent adverse events that were assessed as related by the investigator. The most common were nausea, diarrhea, and upper abdominal pain. Two patients had grade 2 or greater related treatment-emergent adverse events of infection. However, there were no related grade 2 or greater bleeding events, serious adverse events, or deaths.

Chapters:
Introduction 00:00
ITP Overview 00:45
Current Management 2:05
LUNA Clinical Trial 4:44
Take Home Message 8:07
Rilzabrutinib in Other Autoimmune Conditions 9:16

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Catherine M. Broome, MD, Professor of Medicine and Director of Cellular Apheresis, Lombardi Cancer Center, MedStar Georgetown University, discusses real world safety data of Enjaymo (sutimlimab) in patients with cold agglutinin disease (CAD).

CAD is a rare type of autoimmune hemolytic anemia. When the affected individual’s blood is exposed to cold temperatures, IgM antibodies attach to red blood cells and create clumps (agglutination). This causes hemolysis leading to anemia and other associated signs and symptoms. Anemia may cause fatigue, weakness, and pale skin. CAD can be primary or secondary, due to an underlying condition such as an infection, another autoimmune disease, or certain cancers.

Sutimlimab is a humanized monoclonal antibody that specifically binds to complement protein C1s subcomponents in the C1 complement complex. This leads to inhibited activation of the classical complement pathway, preventing deposition of complement opsonins on red blood cells and resulting in inhibition of hemolysis. Sutimlimab is an FDA-approved therapy for hemolysis in adults with CAD.

Recently, real world data on the safety of sutimlimab, was presented at the 2025 American Society of Hematology (ASH) meeting. The data comes from an updated analysis from the CADENCE registry, a multinational, prospective, longitudinal registry for patients with CAD or cold agglutinin syndrome (CAS).

A total of 63 patients were enrolled in the CADENCE registry and treated with sutimlimab between December 12, 2019 and February 4, 2025. This included 58 patients diagnosed with CAD, four with CAS, and one with an unconfirmed diagnosis. At the time of data cut-off, the median and mean sutimlimab treatment duration was 22.7 and 25.1 months, respectively.

A total of 159 adverse events were reported in 52.4% of the sutimlimab-treated patients. 9.5% patients experienced adverse events that the investigator considered related to sutimlimab. Of the 37 serious adverse events reported in 17.5% patients, one serious adverse event, chest pain/stable angina, was considered unexpected and sutimlimab-related. There was one reported death in the cohort, due to low right lung pneumonia, and was considered unrelated to sutimlimab.

Adverse events of special interest in sutimlimab-treated patients included serious infections in three patients, and arterial hypertension in two patients; three patients who experienced adverse events of special interest had concomitantly used rituximab; none of these adverse events of special interest were considered related to sutimlimab. Non-serious adverse events of special interest of acrocyanosis were reported in eight patients, two of which were considered possibly related to sutimlimab.

Chapters:
Introduction 00:00
CAD Overview 00:58
Current Management 2:44
Real-World Safety Analysis 5:13
Take Home Message 8:18

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Anita Hill, MD, PhD, Global Medical Head for Hematology & Nephrology at Alexion, discusses recent data on ravulizumab in rare hematologic disorders.

TMAs are a group of rare disorders that cause blood clots and damage to the walls of the smallest blood vessels in the circulatory system. The blood clots can cause injury to organs that may lead to organ failure and death. HSCT-TMA is a rare and severe type of TMA that occurs following HSCT. A key driver of this disease is terminal complement activation.

PNH is an acquired disorder that leads to the premature death and impaired production of blood cells. People with PNH have recurring episodes of symptoms due to hemolysis, which may be triggered by stresses on the body such as infections or physical exertion. This results in a deficiency of various types of blood cells and can cause signs and symptoms such as fatigue, weakness, abnormally pale skin, shortness of breath, and an increased heart rate. People with PNH may also be prone to infections and thrombosis or hemorrhage, and are at increased risk of developing leukemia. It is caused by acquired genetic changes in the PIGA gene.

Chapters:
Introduction 00:00
HSCT-TMA Data 1:32
Paroxysmal Nocturnal Hemoglobinuria Data 5:12
Safety of Ravulizumab Use in Pregnancy 8:55
Take Home Message for PNH 9:45
Take Home Message for HSCT-TMA 11:10

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Jan Klatt, MD, Head of Development Unit Neurology & Immunology, Merck Healthcare, Germany, discusses the U.S. Food and Drug Administration’s (FDA) Fast Track designation of cladribine capsules for patients with generalized myasthenia gravis (gMG).

gMG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine. The exact reason this occurs is not known.

The U.S. FDA has granted Fast Track designation for cladribine capsules for the treatment of gMG. Fast Track designation is designed to facilitate the development and accelerate the review of therapies for conditions with severe unmet needs. The therapy was previously granted Orphan Drug Designation in 2023.

Cladribine is currently being studied in the global phase 3 MyClad clinical trial (NCT06463587), a global Phase 3, randomized, double-blind and placebo-controlled study designed to assess the safety and efficacy of cladribine capsules in 264 patients with gMG. Cladribine is designed to selectively target B and T cells believed to contribute to the production of harmful autoantibodies that cause inflammation and neuromuscular junction damage.

Results of this trial are expected to be announced upon completion. If approved, cladribine capsules could be the first oral treatment for gMG.

In addition to this designation, collaborations with patient organizations and Ad Scientiam, a medical equipment manufacturing company, to support patient-directed approaches to gMG treatment are ongoing.

To design the MyClad trial and ensure it meets the needs of patients, a patient council was developed and collaborations with over 20 patient advocacy groups around the world were initiated. Insights from the council have informed study protocols, recruitment materials, and potential future product packaging options for the clinical trial program.

The collaboration with Ad Scientiam is also working to launch a prospective, multi-center pilot study exploring an investigational software as a medical device designed to support the management of gMG. The study will investigate how wearable technologies, smartphone-based functional tests, and electronic patient-reported outcomes (ePROs) could detect changes in disease trajectory in real-world conditions.

Chapters:
Introduction 00:00
Myasthenia Gravis Overview 1:05
Cladribine Overview 3:15
MyClad Clinical Trial 5:34
Fast Track Designation 7:54
Patient-Directed Approach 9:33
Take Home Message 12:00

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Floyd Stewart, patient with stage 4 nasopharyngeal carcinoma, and Monique Stewart, advocate and Floyd’s wife, discuss their family’s experience with the disease and their advocacy work.

Nasopharyngeal carcinoma is a rare head and neck cancer arising from the nasopharyngeal epithelium. This type of cancer is rare in the United States, with higher occurrence rates in Asia and Africa. Nasopharyngeal carcinoma is hard to diagnose in the early stages of the cancer since there may be no symptoms at first. Early signs and symptoms may include a lump in the neck caused by a swollen lymph node, bleeding from the nose, bloody saliva, double vision, ear infections, facial numbness, headaches, hearing loss, nasal stuffiness, tinnitus, and sore throat.

Floyd was diagnosed in 2021 after Monique discovered a lump on his neck while the family was on vacation. This diagnosis has been devastating for the couple and their four children. Floyd explains how symptoms, such as sensitive hearing and neuropathy, coupled with the side effects of his treatment, have required the household to adapt to a new way of living.

Floyd also discusses his struggles with feelings of isolation, financial difficulties, and learning how to slow down and listen to his body. Monique also gives insight into all of the emotions that come with being a caregiver, while also a loving wife and mother, explaining finding strength that she never thought she had.

Throughout this journey, the family has grown stronger in their faith and resilience and learned to rely on their community while taking it one day at a time. They stress the importance of not facing these challenges alone and hope that others in similar situations will do the same.

Chapters:
Introduction 00:00
Diagnostic Journey 00:23
Coping With Diagnosis 2:00
How the Family’s Lives Have Changed 3:35
Current Treatment 7:45
Advice for Newly Diagnosed Families 8:23
Importance of Advocacy 10:38

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Vicki Salemi, Career Expert, discusses current trends in the healthcare job market and expectations for 2026.

Recent data from the 2025 Monster Healthcare Market Report shows how the healthcare industry is changing. Through an analysis of healthcare job listings and candidate searches, the report illustrates trends in the healthcare industry shifting to a more digital, distributed, and prevention-focused approach. It also includes insights on where hiring has grown the fastest, and the roles, skills, and credentials predicted to lead the healthcare job market in 2026.

The top 10 most in-demand healthcare jobs in 2025 include:
Registered Nurse
Physical Therapist
Radiology and Imaging Technologists
Licensed Practical/Vocational Nurse
Respiratory Therapist
Occupational Therapist
Speech-Language Pathologist
Certified Nursing Assistant
Nurse Practitioner
Dentist and Allied Dental Professions

Registered nurses and physicians remain as the most in-demand jobs in the field, but growth is emerging in diagnostics, rehabilitation, remote care, and technology-enabled roles. These remote care roles involve specialities in therapy, health information management, pharmaceuticals, and nutrition.

The fastest growing role categories in 2025 included Diagnostic Imaging and Technology, Respiratory and Rehabilitation Services, Nutrition and Wellness, Laboratory and Technical Support, Emergency and Field Care, and Hearing and Speech. Each of these categories grew by at least 30% as compared to 2024.

Data from candidate searches show continuing interest in nursing, coding, and healthcare administration. Job seekers' most-searched healthcare job titles in 2025 included Registered Nurse, Licensed Practical Nurse, Nurse Practitioner, Medical Assistant, Medical Biller, Medical Coder, Healthcare Administration, Medical Office Receptionist, Patient Care Coordinator, and Certified Nursing Assistant.

The top keyword-specific searches from candidates include Epic Analyst/Trainer, Certified Pharmacy Technician (CPhT), CPC (Certified Professional Coder), RBT (Registered Behavior Technician), and LCPC (Licensed Clinical Professional Counselor).
Overall, healthcare continues to be a highly active job market with many opportunities, and demand remains high across all skill levels and experience. In 2026, healthcare demands are expected to expand into new specialties, new settings, and new ways of delivering care.

Chapters:
Introduction 00:00
Top Healthcare Jobs 00:45
Need for Nursing 3:11
Need for Therapists 3:54
Employer’s Needs 4:33
Meeting the Demand Through Recruitment 6:05
Advice for High Demand Professions 7:32
Advice for Employers 9:02

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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 Objectives

• Describe current and emerging gene therapy data in lysosomal disorders and its clinical relevance
• Describe role of patients in gene therapy development

Faculty
Shunji Tomatsu, MD, PhD, Professor and Head, Nemours Children’s Health

Alessandra d’Azzo, PhD, Emerita Faculty, Genetics, St. Jude Children’s Research Hospital

Merve Emecen Sanli, MD, Associate Professor, Department of Pediatrics, University of Texas Southwestern Medical Center

Ryan Colburn. Odimm, Inc.

Disclosures
AffinityCE 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, PhD
Dr. D’Azzo has no relevant financial relationships to disclose.

Merve Emecen Sanli, MD
Dr. Sanli has no relevant financial relationships to disclose.

Ryan Colburn
Mr. 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 Relationships
AffinityCE 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 Designation

Physicians
This 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 Assistants
AffinityCE 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.

Nurses
AffinityCE 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 Practitioners
AffinityCE 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 Counselors
AffinityCE 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 Professionals
All other health care professionals comple...

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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 Objectives
Describe 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.

Faculty
Duarte 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 Medicine

Betul Celik, PhD, Postdoctoral Fellow, Nemours Children’s Health

Gregory Newby, PhD, Assistant Professor, Department of Genetic Medicine, Johns Hopkins School of Medicine

Disclosures
AffinityCE 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, PhD
Dr. Raimundo has no relevant financial relationships to disclose.

Betul Celik, PhD
Dr. Celik has no relevant financial relationships to disclose.

Gregory Newby, PhD
Dr. Newby has no relevant financial relationships to disclose.

Mitigation of Relevant Financial Relationships
AffinityCE 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 Designation

Physicians
This 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 Assistants
AffinityCE 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.

Nurses
AffinityCE 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 Practitioners
AffinityCE 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 Counselors
AffinityCE 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 Professionals
All 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 Costs
There is no cost to participate in this activity.

CME Inquiries
For all CME policy-related inquiries, please con...

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