Scientists on either side of the Atlantic have teamed up, through the new Oxford-Harrington Rare Disease Centre (OHC), to address unmet needs in rare disease research and deliver “major clinical impact” to patients. And now, the international collaboration between the University of Oxford, in England, and the Harrington Discovery…
Researchers have developed a new system that might better measure walking or gait problems in people with Friedreich’s ataxia and other nerve and muscle conditions. The study describing this method was published in the journal Sensors, as “Gauging Gait Disorders with a Method Inspired by Motor Control…
The nonprofit EndFA has launched a free mobile phone and tablet app to help connect, empower, and better inform people with Friedreich’s ataxia (FA) wherever they live. With the mission to “Power the Cure!,” the FA App is designed to connect members of the FA community to each other…
A clinical trial will assess whether supplementation with calcitriol, the active form of vitamin D, can boost frataxin production in people with Friedreich’s ataxia (FA). The Biochemistry of Oxidative Stress Group (BOSG) at the Biomedical Research Institute of Lleida (IRBLleida) and the University of Lleida, in Spain, announced their…
Researchers in Australia were awarded a three-year grant to advance the development of a gene therapy for Friedreich’s ataxia (FA) with the help of stem cells. The grant of $731,061 (AU$982,861) was awarded by the Medical Research Future Fund, and the National Health and Medical Research Council. The research,…
The U.S. Food and Drug Administration (FDA) has given rare pediatric disease and orphan drug designations to LX2006, Lexeo Therapeutics‘ investigational gene therapy for people with Friedreich’s ataxia (FA). The designations each provide benefits meant to ease the therapy’s path through development. Rare pediatric disease status is provided to…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
CRISPR Therapeutics has joined forces with Capsida Biotherapeutics to discover and develop new gene-editing therapies for Friedreich’s ataxia (FA) and familial amyotrophic lateral sclerosis (ALS). The partnership seeks to combine CRISPR Therapeutics’ gene-editing expertise using its proprietary CRISPR/Cas9 platform with Capsida’s tissue-targeted, adeno-associated virus (AAV)-based gene therapies.
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
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