Advocacy

Rare disease advocacy groups amplify the voices of patients, caregivers, and communities to drive awareness, funding, and policy changes. Whether it’s pushing for faster diagnoses, access to treatments, or equitable healthcare policies, advocates play a vital role in transforming lives.
Feb 16, 2025| Posted in: Advocacy, Neurology/Nervous System Diseases

Experiences of Women of Childbearing Age With Epilepsy

Gus Baker, PhD, International Bureau for Epilepsy, discusses…
Feb 15, 2025| Posted in: Advocacy, Neurology/Nervous System Diseases

The Burden of Prolonged Seizures in Patients With Epilepsy

Danya Kaye, UCB Pharma, discusses a study observing…
Feb 4, 2025| Posted in: Advocacy, Cancers, Hematologic Disorders, Treatment

Differences in Clinical Outcomes of Black Patients With Multiple Myeloma

Carolina Schinke, MD, University of Arkansas for Medical…
Jan 31, 2025| Posted in: Advocacy, Congenital And Genetic Conditions, Heart Diseases

Improving Health Equity in Hereditary Angioedema (HAE): A Panel Discussion

This panel discussion by three clinical research leaders…
Jan 17, 2025| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Hematologic Disorders, Treatment

Patient Sentiments Regarding Treatment Options for wAIHA

Nora Sandorfi, MD, Professor of Clinical Medicine, Rheumatology…
Jan 14, 2025| Posted in: Advocacy, Lysosomal Storage Disorders

Gaucher Community Alliance

Madelyn Schloss, Social Media and Development Associate for…

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Join Professors João Gonçalves and Paolo Calicaeti in this educational webinar discussing how PEGylation represents a major technological advancement in the development and optimization of therapeutic proteins.

...https://checkrare.com/optimizing-therapeutic-proteins-through-pegylation/

#CheckRare #RareDisease #lysosomal #RareLysosomal

Optimizing Therapeutic Proteins Through PEGylation: Key Parameters and Impacts (Full Program)

Marina Kremyanskaya, MD, PhD, Icahn School of Medicine at Mount Sinai, discusses the development of gene silencer divesiran for treatment of polycythemia vera (PV).

https://checkrare.com/development-of-gene-silencer-for-treatment-of-polycythemia-vera/

📣 Stay up to date on the most recent FDA approvals and PDUFA dates in the rare disease space with our 2025 Orphan Drugs webpage!

https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/

#CheckRare #RareDiseases #PDUFADates #FDAApproval

This CME program, hosted by John Kuruvilla, MD, explores best practices for discussing possible clinical trial participation with patients who have hematologic malignancies.

#CheckRare #CME

This CME program, hosted by Richard J. Nowak, MD, MS, explains the role of neonatal fragment crystallizable receptor (FcRn) in myasthenia gravis (MG) and how treatments that target FcRn are being used to manage patients with MG.

#CheckRare #CME #MyastheniaGravis

Larisa Geskin, MD, Columbia University Medical Center, discusses the challenges of diagnosing cutaneous T-cell lymphoma.
#CheckRare #RareDisease #CTCL
https://checkrare.com/ctcl-the-role-of-dermatologists-in-diagnosing-and-caring-for-patients/

This CME program, developed by Howard Trachtman, MD, and Carla M. Nester, MD, addresses the complexities involved in diagnosing, treating, and managing patients with various complement-mediated kidney disorders, such as C3 glomerulopathy and atypical hemolytic uremic syndrome (aHUS).

...#CheckRare #CME #RareDisease #KidneyDisorders