by Madaline Spencer and Peter Ciszewski | Jul 27, 2023
Courtney Silverthorn, Ph.D., Associate Vice President for Science Partnerships at the Foundation for the National Institutes of Health (FNIH), discusses the recent announcement that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has...
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...
