POLAR is a randomized, double-blind, placebo-controlled study that enrolled 43 patients with PMM2-CDG across 15 sites ...

Emily Felix, 28, and her family were ‘killed’ by the news that a drug to treat Friedreich’s ataxia may not be considered for ...

POLAR is a randomized, double-blind, placebo-controlled study that enrolled 43 patients with PMM2-CDG across 15 sites globally with topline data expected in the fourth quarter of 2026 Primary endpoint ...

SUNRISE-FA 2 open-label, pivotal trial protocol and SAP for LX2006 submitted to FDA in Q1 2026 following Type B meeting; study initiation on ...

Learn more about whether PTC Therapeutics, Inc. or Relay Therapeutics, Inc. is a better investment based on AAII's A+ ...

Researchers also reported results from a separate trial of a gene therapy the company is developing for PKP2-related arrhythmogenic cardiomyopathy.

CTEXLI approved by FDA for CTX; LIVMARLI approved in Japan for PFIC and Alagille syndrome with partner Takeda; and a single ...

Art therapy is defined by the American Art Therapy Association as utilizing “active art-making, the creative process, and applied psychological theory—within a psychotherapeutic relationship—to enrich ...

Intrahepatic cholangiocarcinoma (iCCA), the second most common primary liver cancer after hepatocellular carcinoma, remains one of the most challenging malignancies to treat due to its highly ...

Carley is a writer, editor and social media professional. Before starting at Forbes Health, she wrote for Sleepopolis and interned at PBS and Nickelodeon. She’s a certified sleep science coach and ...