News

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

The gene therapy startup AavantiBio has launched with $107 million in funding to advance its lead clinical program for the treatment of Friedreich’s ataxia (FA). Early this year, the Boston-based company received a $1 million grant from the Muscular Dystrophy Association (MDA)’s Venture Philanthropy Fund…

The Friedreich’s Ataxia Research Alliance (FARA), the CureFA Foundation, and fara Australia are supporting four research projects that aim to better understand the biology underlying Friedreich’s ataxia (FA) and pave the way for future treatment approaches, FARA announced in a press release. The Award for…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…

The Centers of Medicare & Medicaid Services (CMS) has approved a new, specific diagnosis code for Friedreich’s ataxia (FA) to be used by clinicians in medical records and examined by health insurance companies to determine coverage. “We believe the new FA specific ICD-10 code G11.11 will lead to…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

Ataxia UK has awarded a £26,000 grant to advance research into compounds that can replace or boost the activity of frataxin, the protein that is lost in people with Friedrich’s ataxia (FA), the charity announced in a tweet. Led by Benoit D’Autreaux, PhD, at the…

IXICO has entered a five-year collaboration with the Friedreich’s Ataxia Research Alliance (FARA) and joined the TRACK-FA Neuroimaging Consortium, a group of academic and industry partners that are conducting a natural history study focused on tracking brain and spinal cord changes in patients with Friedreich’s ataxia (FA).

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

People with Friedreich’s ataxia (FA) are being urged to join the Friedreich’s Ataxia Global Patient Registry (FAGPR) to help advance knowledge into disease progression and support treatment development. Managed by international advocacy organizations, the patient registry seeks to collect data from volunteers worldwide to improve clinical trial…