Rare and Genetic Disease Network

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Orphan Drug Development, Pricing, and Access

Dennis Jackman, Senior Vice President, Public Affairs at CSL Behring discusses rare disease drug pricing, patient involvement in the drug development process, and drug access.

Thyroid Eye Disease: Incidence and Treatment

Gary Joseph Lelli, MD, of the Department of Ophthalmology at Weill Cornell Medicine talks about the incidence and treatment options for thyroid eye disease due to Graves’ disease.

Hemophilia Alliance: The Original Managed Care Plan

Joe Pugliese discusses the Hemophilia Alliance, a not-for-profit organization of federally funded hemophilia treatment centers that is working to ensure its member have the expertise, resources and public support to sustain their integrated clinical and pharmacy services for persons with hemophilia and other bleeding disorders.

Tenosynovial Giant Cell Tumor (TGCT)

Tenosynovial giant cell tumor (TGCT) is a benign tumor involving the joint synovium, bursae, and tendon sheath. These rare tumors are sometimes referred to as giant cell tumor of the tendon sheath (GCT-TS) and/or pigmented villonodular synovitis (PVNS).

Patient Advocacy Certificate Training

David Pearce, PhD, of the Coordination of Rare Diseases at Sanford (CoRDS) talks about their patient advocacy conference held in Sioux Falls, SD.

An Oral Film to Treat Lennox-Gastaut Disease

Daniel Barber is chief operating officer at Aquestive Therapeutics, a specialty pharmacy company that is highly focused on providing treatment options for persons unable to properly swallow medicine (e.g., people with epilepsy, ALS, Parkinson’s, etc).

FDA Reviewing Drug Application for Neuromyelitis Optica

The U.S. Food and Drug Administration (FDA) has accepted Genentech’s Biologics License Application (BLA) for satralizumab to treat of adults and adolescents with neuromyelitis optica spectrum disorder (NMOSD).

Diagnosing Congenital Hyperinsulinism

Julie Raskin, Executive Director of Congenital Hyperinsulinism International (CHI) explains how congenital hyperinsulism is diagnosed and the latest research is underway to improve how it is diagnosed and understood.

Special Reports

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