News

‘Spennylympics’ Duo in UK Raise £150,000 for MND Association

Completing their version of 102 Olympic events — including archery, diving, and fencing — in 17 days, Stuart Bates and Charlotte Nichols raised some £150,000 (about $207,000) for the U.K.’s Motor Neurone Disease (MND) Association. The Oxfordshire duo wound up their grueling challenge, thought to be the first of…

Register Now for Global Genes’ RARE Patient Advocacy Summit

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

ALS Cellucci Fund Marathoners Hope to Raise $50K

It’s been a decade since the first UMass ALS Cellucci Fund team first participated in the Boston Marathon to raise funds to support University of Massachusetts Medical School (UMMS) researchers in their quest to find better treatments for amyotrophic lateral sclerosis (ALS). So, this year is significant. The…

TP73 Gene Mutations May Be Risk Factor for Sporadic ALS

Rare mutations in the TP73 gene, which is involved in the regulation of a cell’s life cycle, may put a person at greater risk of amyotrophic lateral sclerosis (ALS), according to a genetic analysis of nearly 2,900 sporadic ALS patients. Such mutations were found to affect the maturation and survival of…

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

Ketamine Earns FDA’s Orphan Drug Designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ketamine, PharmaTher’s investigational therapy for amyotrophic lateral sclerosis (ALS). Orphan drug status is intended to encourage the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. It provides…