by Madaline Spencer | Oct 4, 2024
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare autoimmune lung disorder. It is the most common form of pulmonary alveolar proteinosis. Etiology aPAP is caused by an immune system malfunction, due to IgG antibodies that block the...
by Madaline Spencer | Oct 3, 2024
Kurt Gunter, MD, Senior Vice President and Chief Medical Officer at Inozyme Pharma, discusses data presented on ENPP1 deficiency and ABCC6 deficiency at the 2024 American Society for Bone and Mineral Research meeting. ENPP1 ENPP1 deficiency is a rare...
by Madaline Spencer | Oct 1, 2024
Paul Bolno, CEO, and Anne-Marie Li-Kwai-Cheung, Chief Development Officer, of Wave Life Sciences, discuss interim data from the ongoing FORWARD-53 clinical trial for Duchenne muscular dystrophy (DMD). DMD affects the muscles, leading to progressive...
by Madaline Spencer | Sep 30, 2024
Tiffany Chow, MD, Behavioral Neurologist, Vice President of Clinical Development at Alector, discusses a survey conducted to better understand caregivers’ experiences With frontotemporal dementia (FTD). FTDs are a group of neurodegenerative disorders...
by Madaline Spencer | Sep 26, 2024
Andrew Dauber, MD, Chief of Endocrinology at Children’s National Hospital, discusses data presented on the efficacy of vosoritide in genetic skeletal conditions. Disease Overview Achondroplasia is a rare skeletal disorder that prevents the changing of cartilage...
