The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, voted today to add spinal muscular atrophy (SMA) to the national list of disorders recommended for screening at birth. Following today’s vote, the Secretary of Health and Human Services (HHS) will make a final decision on whether . . .
PTC Therapeutics reported today the U.S. Food and Drug Administration’s Office of Drug Evaluation is unable to approve the company’s New Drug Application for ataluren (brand name Translarna) for the treatment of Duchenne muscular dystrophy (DMD) caused by a premature stop codon, or nonsense mutation, in its current form.
“It truly feels as if my entire career path has been leading me to this role,” said Vos. “I am excited to bring my passion for patient care and advocacy, as well as my success using new technology to build new, innovative communication approaches, to lead MDA in the fast-changing world of research and patient care.”
After the news of MDA champion Jerry Lewis’s passing broke Sunday, scores of admirers took to social media to remember the comedian and philanthropist. Here are just a few of the thousands of tweets sent by some of the entertainment business’ brightest stars — and some of MDA’s staunchest partners. Want to add your remembrance to the chorus? Visit our Tributes page, where we are collecting memories for Jerry’s family.
Today, on the first Friday of ALS Awareness Month, the U.S. Food and Drug Administration (FDA) approved edaravone (brand name Radicava), to treat ALS (amyotrophic lateral sclerosis). Under development by Mitsubishi Tanabe Pharma America, Radicava was approved in 2015 to treat ALS in Japan. It’s the first drug to be granted FDA approval to treat ALS in the United States in more than 20 years.