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...
by Madaline Spencer | May 14, 2024
Patricia Weltin, CEO of Beyond the Diagnosis, provides an overview of tethered spinal cord syndrome (TSCS). Tethered spinal cord syndrome is a progressive, nervous system disorder in which tissue attaches to the spinal cord. This limits movement and...
