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FDA Approves Nerandomilast To Treat Patients With Idiopathic Pulmonary Fibrosis

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...

ISUOG World Congress 2025: Hemolytic Disease of the Fetus and Newborn

by Madaline Spencer | 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...

Neuroblastoma: New Approaches to Neuroblastoma Consortium

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...

Results From the PEGASUS Clinical Trial of Pegvaliase in Patients With PKU

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...

Plans for Phase 2/3 Clinical Trial of Bexmarilimab Plus Standard of Care in Patients With Myelodysplastic Syndromes

by Madaline Spencer | Oct 6, 2025

Amer Zeidan, MBBS, MHS, Professor of Internal Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center, discusses plans for a phase 2/3 clinical trial testing bexmarilimab plus standard of care in patients with...
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