News

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

Nominations Open for 2022 Eurordis Black Pearl Awards

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

Study Will Test Using Calcitriol to Boost Frataxin

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…

Stem Cell Therapy Researchers in Australia Awarded 3-Year Grant

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,…

Potential Gene Therapy for Heart Disease in FA Gets FDA Support

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…

CRISPR Partners with Capsida to Develop Gene-editing Therapies

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.

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

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…