News

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,…

People with Friedreich’s ataxia (FA) are encouraged to enroll in three clinical trials evaluating the effects of dietary supplements on exercise, the safety and efficacy of the experimental therapy vatiquinone, and the utility of wearing sensors to monitor the disease’s progression and severity. The Friedreich’s Ataxia Research Alliance…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Truncated forms of frataxin, the protein that is lost in people with Friedreich’s ataxia (FA), were found predominantly in mouse tissues, contrasting with the dominance of non-truncated frataxin in human tissue, a study found. The findings have implications for mouse models used to mimic the condition in disease research…

The U.S. Food and Drug Administration has put a hold on the clinical development of CTI-1601, an investigational treatment for Friedreich’s ataxia (FA) that is being developed by Larimar Therapeutics. According to a press release from Larimar, the hold was put in place after Larimar notified the…

The U.S. Food and Drug Administration (FDA) has asked Reata Pharmaceuticals to request a preliminary meeting to discuss submitting a new drug application (NDA) for omaveloxolone, an investigational therapy for Friedreich’s ataxia (FA). Reata was due to hold a Type C meeting (unrelated…