News

Healx has partnered with the Friedreich’s Ataxia Research Alliance (FARA) and Ataxia UK to combine its drug discovery expertise with the organizations’ patient and scientific insights in order to develop potential treatments for Friedreich’s ataxia. “We believe that partnership and collaboration — sharing knowledge and resources —…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Stealth BioTherapeutics is planning a Phase 2a clinical trial of elamipretide (SS-31) as a treatment for people with vision loss and/or heart disease associated with Friedreich’s ataxia (FA). The company expects to use data from this study — planned to start later this year — in designing a pivotal…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Six months of treatment with interferon-gamma, known as IFN-gamma, is safe and leads to significant improvements in walking skills and stance in people with Friedreich’s ataxia (FA), according to a small study in Turkey. However, no significant improvements were observed in other areas of motor coordination, such as sitting, upper…

Lexeo Therapeutics has entered an agreement with Adverum Biotechnologies to license Adverum’s intellectual property rights and pre-clinical data of LX2006, an investigational gene therapy for Friedreich’s ataxia (FA). Lexeo will continue to advance the gene therapy program through pre-clinical studies that support an investigational new drug (IND) application,…

GeneDx has expanded its list of genetic tests to include the analysis of expansion repeats that underlie adult-onset ataxias, including Friedreich’s ataxia (FA), spinocerebellar ataxia, and other forms of inherited ataxias. With this expansion, GeneDx — a subsidiary of BioReference Laboratories, part of the OPKO Health company —…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease and fast track designations to Retrotope’s investigational therapy RT001 for the treatment of Friedreich’s ataxia (FA), the company announced in a press release. RT001 also received orphan drug designation from the European Medicines Agency (EMA)…