Forest Ray PhD,  —

A Phase 1 clinical trial of a gene-based therapy for amyotrophic lateral sclerosis (ALS) is now recruiting patients in one of its European centers. The Clinical Research Facility at St James’s Hospital Dublin, in Ireland, has enrolled its first patient and is one of four European centers to participate…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

The U.S. Food and Drug Administration has granted Prosetin orphan drug designation for the treatment of amyotrophic lateral sclerosis (ALS). Orphan drug designation grants financial incentives to companies developing medications for rare diseases, which might not be profitable enough to pursue without government assistance. While a Phase…

Accessing and understanding insurance coverage, and paying for medical treatments and services top the list of stressors that burden people with amyotrophic lateral sclerosis (ALS) in the United States, a survey of patients and caregivers reports. Ten percent of its 444 respondents — in 1 in every 10 —…

A new telehealth app to help people with motor neuron diseases such as amyotrophic lateral sclerosis (ALS) connect with their healthcare providers during the COVID-19 pandemic has been fast-tracked for use in the United Kingdom. The Telehealth in Motor Neurone Disease (TiM) system, developed by the  …

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Innovate UK has given a grant to Reflection Therapeutics to help fund research into new ways of protecting the nervous system from motor neuron diseases, with amyotrophic lateral sclerosis (ALS) as an initial focus. The Innovate Smart Grant, which is reported to be…

The greater incidence of amyotrophic lateral sclerosis (ALS) among professional athletes and people with head trauma, and the faster functional decline seen in animal models after localized nerve injury, strongly suggests that nerve injury plays a role in initiating disease processes, a review study reported. Needed avenues of future…

For three weeks during Lou Gehrig’s final season, the baseball legend appeared to shake off his symptoms of amyotrophic lateral sclerosis (ALS) and regain his flagging prowess. Richard Bedlack, MD, PhD, founder and director of Duke University’s ALS clinic, and baseball author Dan Joseph, provide an…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…