by Peter Ciszewski | Nov 30, 2018
Some long-awaited good news arrived for the Lambert-Eaton Myasthenic syndrome (LEMS) community this week as the U.S. Food and Drug Administration approved Firdapse (amifampridine) tablets for the treatment of LEMS in adults. LEMS is a rare autoimmune disorder that...
by Peter Ciszewski | Nov 28, 2018
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, discusses how his company works with Dravet syndrome advocacy groups, including the Dravet Syndrome Foundation (DSF). The DSF raises research funds for Dravet syndrome and related epilepsies. The...
by Peter Ciszewski | Nov 28, 2018
The U.S. Food and Drug Administration granted accelerated approval to Vitrakvi (larotrectinib, Loxo Oncology and Bayer) for the treatment of patients with solid tumors who have an NTRK gene fusion without a known resistance mutation. This approved indication applies...
by Peter Ciszewski | Nov 27, 2018
Richard A. Basile Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses his organization’s business model, focus on rare diseases, and willingness to partner with patients, academic centers, investors, and volunteers. BioPontis is an...
by Peter Ciszewski | Nov 24, 2018
Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses Krabbe disease (also called globoid cell leukodystrophy), a degenerative disorder that affects the nervous system. It is caused by...
