by Madaline Spencer | Mar 29, 2024
Alex Kolevzon, MD, Clinical Director of the Seaver Autism Center at Mount Sinai, discusses Phelan-McDermid syndrome and the JAG201 study. Phelan-McDermid syndrome is a rare, genetic form of autism spectrum disorder. It is caused by a variant in the...
by Madaline Spencer | Mar 29, 2024
The U.S. Food and Drug Administration has approved WINREVAIR (sotatercept-csrk) for the treatment of pulmonary arterial hypertension (PAH). PAH is a rare disorder affecting the heart and lungs. It is characterized by abnormally high blood pressure...
by Madaline Spencer | Mar 27, 2024
The U.S. Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz) for the treatment of adult patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) that tests positive for anti-aquaporin-4 (AQP4 Ab+). This approval is a major milestone as it...
by Madaline Spencer | Mar 27, 2024
Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases. The study, “Measurement of glycosaminoglycans in the amniotic fluid of fetuses with mucopolysaccharidoses treated in a...
by Madaline Spencer | Mar 25, 2024
F. Sessions Cole, Secretary, UDNF and Professor of Pediatrics, Washington University School of Medicine, and Michele Herndon, MSN, RN and Program Director of UDNF’s Patient Navigation Program, discuss the Undiagnosed Diseases Network Foundation and their Patient...
