by Madaline Spencer and Peter Ciszewski | Jul 20, 2023
Rennie McCarthy, Chief Executive Officer at Stealth Biotherapeutics, provides an overview of mitochondrial myopathy, or myopathic mitochondrial DNA depletion syndrome. Mitochondrial myopathy is a rare genetic disorder caused by mutations in nuclear...
by Madaline Spencer and Peter Ciszewski | Jul 11, 2023
Kim Moran, Ph.D., MBA, Head of US Rare Diseases at UCB, discusses the use of phish finder programs to find undiagnosed patients. AI and data mining programs can help companies find persons with early symptoms of diseases based on clinical records....
by Madaline Spencer and Peter Ciszewski | Jul 4, 2023
Kim Moran, Ph.D., MBA, Head of US Rare Diseases at UCB, discusses the two orphan drugs, rozanolixizumab and zilucoplan, which the company hopes to have approved shortly to treat myasthenia gravis. Myasthenia gravis is a rare autoimmune disorder that targets the...
by Peter Ciszewski | Jun 30, 2023
The U.S. Food and Drug Administration (FDA) has approved BioMarin’s gene therapy [valoctocogene roxaparvovec-rvox (Roctavian)] to treat adults with severe hemophilia A. This is an adeno-associated virus vector (AAV)–based gene therapy and will be a one-time infusion...
by Peter Ciszewski | Jun 28, 2023
Nicholas Johnson, MD, Associate Professor in the Department of Neurology at Virginia Commonwealth University, explains myotonic dystrophy type 1 and how it is currently managed. DM1 is a progressive neuromuscular disease caused by a triple repeat in the DMPK...
