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...
by Peter Ciszewski | Nov 18, 2019
The U.S. Food and Drug Administration (FDA) has approved Brukinsa (zanubrutinib) for treating adults with mantle cell lymphoma (MCL) who have received at least one prior therapy. This is the first drug to be FDA approved by the Chinese based company, BeiGene Ltd. The...
