by Madaline Spencer | Apr 29, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni (lunsotogene parvec) for the treatment of patients with otoferlin (OTOF)-related hearing loss. The indication includes pediatric and adult patients with severe-to-profound and...
by Madaline Spencer | Apr 29, 2026
A study published in the Journal of Clinical Medicine evaluated the improvement of bone mineral density in patients with type 1 Gaucher disease (GD1) treated with velaglucerase alfa. GD1 is the most common form of Gaucher disease, characterized by a deficiency of...
by Madaline Spencer | Apr 28, 2026
Arthur Beisang, MD, Department of Pediatrics at Gillette Children’s Specialty Healthcare in Saint Paul, Minnesota, discusses Daybue (trofinetide) Stix, a new formulation of the treatment for Rett syndrome. Rett syndrome is a neurodevelopmental condition...
by Madaline Spencer | Apr 27, 2026
A study recently published in Health and Quality of Life Outcomes analyzed quality of life in patients with pulmonary arterial hypertension (PAH). PAH is a rare condition affecting the heart and lungs. It is characterized by abnormally high blood pressure...
by Madaline Spencer | Apr 24, 2026
Richard J. Nowak, MD, global principal MINT investigator and director of the Myasthenia Gravis Clinic at Yale University, discusses a post-hoc analysis of Uplizna (inebilizumab) on the effect of ocular manifestations in generalized myasthenia gravis (gMG). gMG...
