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...
by Madaline Spencer | Apr 23, 2026
Fernanda Leal-Pardinas, MD, MSc, Global Clinical Development Lead, Rare & Metabolic Disorders at Otsuka Pharmaceutical, discusses the open-label extension data from their study testing repinatrabit for patients with phenylketonuria (PKU). PKU is a...
by Madaline Spencer | Apr 22, 2026
A retrospective observational study published in the International Journal of Cardiology evaluated a new clinical staging of hypertrophic cardiomyopathy in Fabry disease. Fabry disease is a lysosomal disorder caused by mutations in the GLA gene. It is characterized by...
