by Madaline Spencer | Sep 28, 2026
Amy Waldman, MD, pediatric neurologist and Medical Director of the Leukodystrophy Center at the Children’s Hospital of Philadelphia, discusses the approval of Zanvastro (zilganersen) to treat patients with Alexander disease. Alexander disease is a rare...
by Madaline Spencer | Sep 25, 2026
A study published in The New England Journal of Medicine examined results from the phase 3 TETON-1 clinical trial of inhaled treprostinil for idiopathic pulmonary fibrosis (IPF). IPF is a rare, progressive disorder characterized by the progressive scarring of tissues...
by Madaline Spencer | Sep 23, 2026
A study published in Pulmonary Circulation evaluated the association between pulmonary arterial hypertension (PAH) diagnosis and risk of newly diagnosed mental health conditions. PAH is a rare condition affecting the heart and lungs, characterized by abnormally high...
by Madaline Spencer | Sep 22, 2026
The US Food and Drug Administration (FDA) has approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in patients with ataxia-telangiectasia who weigh at least 33 pounds (15 kg). It is the first treatment approved for this rare condition. Ataxia...
by Madaline Spencer | Sep 22, 2026
Jenny McCue, Vice President of Global Research and Clinical Development at the Myasthenia Gravis Foundation of America (MGFA), discusses research highlights to be presented at the upcoming 2026 MGFA Scientific Session being held September 29th in Orlando, FL. ...
