by Madaline Spencer | May 21, 2024
Alan Percy, MD, pediatric neurologist at University of Alabama at Birmingham, discusses the DAFFODIL clinical trial, testing the safety and tolerability of trofinetide for the treatment of Rett syndrome. Rett syndrome is a neurodevelopmental condition...
by Madaline Spencer | May 17, 2024
Doug Treco, PhD, CEO of Inozyme Pharma, discusses ongoing clinical trials for INZ-701, an enzyme replacement therapy for the potential treatment of ENPP1 and ABCC6 Deficiencies. ENPP1 Deficiency and ABCC6 Deficiency are rare metabolic diseases caused by...
by Madaline Spencer | May 17, 2024
Alan Percy, MD, Pediatric Neurologist at University of Alabama at Birmingham, discusses the current management and obstacles of Rett syndrome patients. Rett syndrome is a neurodevelopmental condition primarily affecting girls. Patients with the disease...
by Madaline Spencer | May 16, 2024
James Howard, MD, Neurologist at University of North Carolina at Chapel Hill, discusses the current and emerging treatment options of myasthenia gravis. Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the...
by Madaline Spencer | May 15, 2024
Doug Treco, PhD, CEO of Inozyme Pharma, provides an overview of ENPP1 Deficiency and ABCC6 Deficiency. ENPP1 ENPP1 Deficiency is a rare metabolic disease caused by mutations in the ENPP1 enzyme. This enzyme is responsible for the cleavage of...
