While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…
Expecting to award roughly $18 million this year in grants to support research in neuromuscular diseases, the Muscular Dystrophy Association (MDA) continues to be a leader in efforts to better treat and otherwise ease life for those touched by Friedreich’s ataxia as well as muscular dystrophy and other,…
Gene therapies — after a tragedy kicked them back to the lab two decades ago — are beginning to come into their own, making possible the progress in gene-targeted and combination treatments for neuromuscular diseases once thought unimaginable. But many challenges remain — from the likelihood of no “one and…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
Dr. Colin Meyer, the chief medical officer and vice president of Reata Pharmaceuticals, spoke Friday in a taped interview with reporter Patricia Inacio about omaveloxolone, an oral therapy in line to possibly become the first FDA-approved treatment for Friedreich’s ataxia. A Phase 2 clinical trial, called MOXIe (NCT02255435), is moving into a second and…
Intimate and focused, conferences like IARC 2017 — the International Ataxia Research Conference — are the type of scientific meeting that “all rare disease patient groups should try to do,” Warren Huff, the founder and chief executive officer of Reata Pharmaceuticals, said in an interview with Friedreich’s Ataxia…
In this Facebook Live Chat, reporter Hal Foster — joined by two leading Friedreich’s ataxia researchers, Louise Corben with the Murdoch Childrens Research Institute in Australia, and David Lynch with Children’s Hospital of Philadelphia — looked back at day three of IARC 2017 in Pisa, Italy, and toward what the future might…
In this Facebook Live Chat, reporter Hal Foster — joined by scientists and leaders of groups working to promote research and patient interests in Friedreich’s and other ataxias — looks back at highlights of sessions and other happenings on the second day of IARC 2017 in Pisa, Italy. IARC is the world’s largest…
In this Facebook Live Chat, reporter Hal Foster — joined by scientists and leaders of groups working to promote research and patient interests in Friedreich’s and other ataxias — looks back at highlights of the opening day of IARC 2017 in Pisa, Italy. …
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