Surgery for Thyroid Eye Disease

  Gary Joseph Lelli, MD, of the Department of Ophthalmology at Weill Cornell Medicine discusses surgical options currently available to treat thyroid eye disease due to Graves’ disease. Graves’ disease is an immune system disorder that results in the...

Hemorrhagic Stroke

  Rajiv Ratan, MD, PhD, of the Burke Neurological Institute, explains hemorrhagic stroke. A hemorrhagic stroke occurs when a blood vessel bursts and bloof leaks into the surrounding brain tissue. Surprisingly, this type of stroke is relatively rare (compared to...

Partnering with Rare Disease Groups

  David Pearce, PhD of Sanford Research discusses how organizations can partner in rare diseases. That is the issue that is being addressed by a number of organizations, including Sanford Research and the International Rare Disease Research Consortium – two...

FDA approves Givosiran to Treat Rare Liver Condition

The U.S. Food and Drug Administration (FDA) has approved Givlaari (givosiran) to treat acute hepatic porphyria, a rare genetic disorder that causes a buildup of porphyrins in the body. Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and...

Friedreich’s Ataxia

  Brigid Brennan of the Friedreich’s Ataxia Research Alliance (FARA) discusses Friedreich’s ataxia (FA), a degenerative neuro-muscular disorder due to mutations in the FXN gene that reduces the production of frataxin, a protein important for proper mitochondrial...

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. With regard to the latter issue, Jackman said, “if the community and industry,...
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