News

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

74,000 Sign FARA Petition Asking FDA, Reata to Move Ahead on Omav

Over about two weeks, more than 74,000 people worldwide have signed a petition from the Friedreich’s Ataxia Research Alliance (FARA) requesting that omaveloxolone (omav) be made available to people with Friedreich’s ataxia (FA) as quickly as possible. The petition specifically asks Reata Pharmaceuticals to submit a new drug…

Leriglitazone Shows Benefits in Cell and Animal Models

Leriglitazone, an oral treatment candidate for Friedreich’s ataxia (FA), can increase frataxin levels and reduce neuronal degeneration in cell and animal models of the disease, a study found. Along with promising clinical findings from a Phase 2 clinical trial (NCT03917225), the results support the use of leriglitazone, and the…

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Startup Lexeo Raises $85M to Pursue 3 New Gene Therapies

Lexeo Therapeutics recently launched with an initial funding of $85 million to develop gene therapies for Friedreich’s ataxia (FA) and other disorders. The proceeds will support the clinical development of three gene therapy programs, including an anticipated Phase 1 trial this year to test LX2006 for FA-associated heart…

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Center of Excellence Awarded Nearly $1.3M to Advance Research

The Friedreich’s Ataxia Center of Excellence of the Children’s Hospital of Philadelphia (CHOP) has been awarded a total of $1.275 million to advance research and clinical care of people with Friedreich’s ataxia (FA). The grant came from the Friedreich’s Ataxia Research Alliance (FARA), the Hamilton and Finneran families, and…

NORD’s Caregiver Respite Program Continues Through Pandemic

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…