by Madaline Spencer | Mar 31, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-1 (LAD-1). The indication is for patients with biallelic variants in...
by Madaline Spencer | Mar 31, 2026
Joanne Donavan, MD, PhD, Chief Medical Officer at Edgewise Therapeutics, discusses the MESA extension study of sevasemten for the treatment of patients with Becker muscular dystrophy (BMD). BMD is a genetic condition characterized by a lack of working...
by Madaline Spencer | Mar 30, 2026
A study published in The Cureus Journal of Medical Science analyzed a case study of an adolescent with Rapid Eye Movement (REM) sleep behaviour disorder in Moebius syndrome. Moebius syndrome is a rare neurological condition that primarily affects the muscles that...
by Madaline Spencer | Mar 30, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval to Avlayah (tividenofusp alfa) for the treatment of neurologic manifestations of Hunter syndrome (MPS II). This is the first FDA-approved transferrin receptor (TfR)-enabled therapeutic...
by Madaline Spencer | Mar 27, 2026
Paul Romness, CEO of OS Therapies, discusses the Plausible Mechanism Framework draft guidance and how it will affect the approval process of OST-HER2 LM for the treatment of osteosarcoma. Osteosarcoma is the most common type of bone cancer, typically occurring...
