A baby from Cambridge was born with a syndrome so rare it has no name after doctors noticed his “beautiful” wide eyes. Oska-Gray Valentine-Colquhoun spent his first days in the NICU and needed ...

BOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare ...

Department of Medical Biology, Faculty of Health Sciences, Medical University of Warsaw, Warsaw, Poland Department of Interdisciplinary Research in Social Inclusion, Institute of Special Education, ...

Ionis Pharmaceuticals, which is developing a treatment for the rare neurological disease of Angelman syndrome (AS), turned to Lifetime Television’s “Behind the Mystery” series to increase public ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced top-line results from the Phase 3 COMPASS PWS trial evaluating the efficacy and safety of intranasal carbetocin ...

Soleno Therapeutics is breathing easier with a report from Acadia Pharmaceuticals that its candidate to treat hyperphagia in patients with Prader-Willi Syndrome (PWS) has come up short in a phase 3 ...

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...

The Cubs have landed utilityman Willi Castro from the Twins, according to Jon Heyman of the New York Post. Minnesota will receive multiple prospects in return, and ESPN.com’s Jesse Rogers specifies ...

The Astros’ desire to add some left-handed balance to their righty-heavy lineup doesn’t mean the team is restricting itself to solely lefty-swingers. Houston has interest in switch-hitting utilityman ...