Mary Chapman,  features writer—

The Institute for Gene Therapies (IGT) has launched, with the aim of maximizing the potential of gene therapies in genetic disorders such as Friedreich’s ataxia (FA). Comprised of industry leaders, scientists, and patient advocates, the IGT’s overarching goal is to set the foundation for a modernized regulatory…

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Kevin Schaefer hadn’t been in an airport since he was 4 years old, so he had been looking forward to flying from his home in Cary, North Carolina, to Anaheim, California, in June for the 2019 Cure SMA Conference. As it turned out, his experience didn’t go as expected.

The Muscular Dystrophy Association (MDA) has awarded AavantiBio $1,076,232 to advance the biotechnology company’s development of a gene replacement therapy for Friedreich’s ataxia (FA). Awarded through the MDA Venture Philanthropy drug development program, the funds will help launch production of the clinical gene vector…

Online forums have markedly altered how patients, caregivers and healthcare professionals communicate and connect. Even when miles apart, individuals can establish supportive relationships, share experiences and information, and sometimes simply vent. Bionews Services, a leading online health, science and research publication company, has been rolling out its…

A new data-sharing platform recently launched by the Critical Path Institute’s (C-Path) Data Collaboration Center (DCC) and the Friedreich’s Ataxia Research Alliance (FARA) is designed to ultimately lead to the development of effective treatments for Friedriech’s ataxia (FA). Called the Friedreich’s Ataxia Integrated Clinical…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

Neurocrine Biosciences and Voyager Therapeutics have formed an alliance in hopes of bringing potentially life-changing treatments to market for Friedreich’s ataxia (FA) and Parkinson’s disease. The collaboration is aimed at developing and commercializing Voyager’s gene therapy programs, including VY-FXN01 for Friedreich’s and VY-AADC for…

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…