by Peter Ciszewski | Nov 22, 2019
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...
by Peter Ciszewski | Nov 21, 2019
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...
by Peter Ciszewski | Nov 20, 2019
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...
by Peter Ciszewski | Nov 20, 2019
The U.S. Food and Drug Administration (FDA) approved Adakveo (crizanlizumab-tmca) to reduce the frequency of pain crises in individuals with sickle cell disease. Adakveo (Novartis) is indicated for patients ages 16 or older is a humanized anti-P-selectin monoclonal...
by Peter Ciszewski | Nov 19, 2019
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...
