by Madaline Spencer | Oct 10, 2025
Mike Flanagan, PhD, Chief Scientific Officer at Avidity Biosciences, discusses new data from the EXPLORE44 open-label extension of del-zota for treatment of patients with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping. DMD affects the...
by Madaline Spencer | Oct 9, 2025
The U.S. Food and Drug Administration (FDA) has approved Jascayd (nerandomilast) tablets for the treatment of patients with idiopathic pulmonary fibrosis (IPF). This is the first new therapy to be approved in this indication for 10 years. IPF is a rare, progressive...
by Madaline Spencer and Joe Haddad | Oct 9, 2025
Jannine Williams, Compound Development Team Leader at Johnson & Johnson, discusses key takeaways from studies on hemolytic disease of the fetus and newborn (HDFN) presented at ISUOG World Congress 2025. HDFN is a rare condition characterized by the...
by Madaline Spencer | Oct 8, 2025
Araz Marachelian, MD, Pediatric Oncologist at Children’s Hospital Los Angeles, discusses the New Approaches to Neuroblastoma (NANT) Consortium and its work in neuroblastoma. Neuroblastoma is a rare cancer that largely afflicts infants or children. It...
by Madaline Spencer | Oct 7, 2025
Kevin Eggan, PhD, Chief Scientific Officer at BioMarin, discusses new results from the PEGASUS clinical trial of Palynziq (pegvaliase) for treating patients with phenylketonuria (PKU). PKU is a genetic metabolic disorder that increases the body’s...
