The Friedreich’s Ataxia Research Alliance (FARA)Â has announced the 2016 winners of the Ataxian Athlete Initiative (AAI), a unique…
Articles by Margarida Azevedo, MSc
The Friedreich’s Ataxia Research Alliance (FARA) joined the California NAACP and other patient advocacy groups to listen to opinions and concerns about SB…
Jupiter Orphan Therapeutics Inc. (JOT)Â announced that David Sinclair, a Harvard Medical School professor with an interest in the dietary…
The U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development recently granted orphan drug designation to Retrotope‘s stabilized…
Global Genes, a leading rare genetic disease advocacy organization, has just announced the winners of its inaugural RARE…
Agilis Biotherapeutics, LLC, and Waisman Biomanufacturing have signed an exclusive partnership agreement to develop and produce AGIL-FA, Agilis’ potential gene therapy…
University of Texas (UT)Â Southwestern Medical Center researchers have identified RNA and DNA synthetic agents that increase frataxin levels and alleviate…
Retrotope announced the completion of a first dosing cohort, and the opening of patient enrollment for a highest dose cohort, in its…
Researchers have demonstrated in the lab (in vitro) and in living subjects (in vivo), that gene transfer of brain-derived neurotrophic factor (BDNF)…
RaNA Therapeutics, a Cambridge, Massachusetts-based biotech company that develops next-generation RNA-targeted medicines and which currently has a lead program in…