by Peter Ciszewski | Aug 5, 2019
David Anderson, PhD of Spark Therapeutics is helping the company develop a gene therapy for Batten disease. In this clip, Dr. Anderson describes this rare disease and the need for a gene therapy to cure this progressive, neurodegenerative disorder. Batten...
by Peter Ciszewski | Aug 4, 2019
The U.S. Food and Drug Administration (FDA) granted approval to Turalio (pexidartinib) capsules for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not responsive to...
by Peter Ciszewski | Aug 2, 2019
Andra Stratton, President and co-founder of Lipodystrophy United talks about the foundation and the large increase in individuals being diagnosed with the disease, possibly as a result of the work LU has done to raise awareness of this rare condition. Stratton...
by Peter Ciszewski | Aug 1, 2019
Recently we talked with Dennis Jackman, senior vice-president of public affairs at CSL Behring about the company’s rare disease orphan drug pipeline. “First of all, we have existing therapies and we’re trying to find new uses for those therapies for rare...
by Peter Ciszewski | Jul 31, 2019
John Maslowski is President and Chief Executive Officer (CEO) of Fibrocell Science, a biotech company that is developing cell and gene therapies for a variety of rare, mostly skin-related, conditions. At the BIO International Convention held in Philadelphia,...
