News

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

I AM ALS, a nonprofit working for people affected by amyotrophic lateral sclerosis (ALS), is offering a guide to help others who want to follow in its footsteps — creating a movement that mobilizes and unites a community against a disease, it announced in a press release. This free…

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…

Existing safety and effectiveness data from a Phase 3 clinical trial of NurOwn — an investigational cell-based therapy for amyotrophic lateral sclerosis (ALS) — are not sufficient to support the therapy’s approval, the U.S. Food and Drug Administration (FDA) concluded in an initial review. According to the…

The My Name’5 Doddie Foundation has donated a total of £300,000 (about $420,600) to two U.K. nonprofits to support people living with motor neuron diseases (MND), including amyotrophic lateral sclerosis (ALS). The foundation, established by the former Scottish rugby player George Wilson “Doddie” Weir, gave £200,000 to the…

Military veterans with amyotrophic lateral sclerosis (ALS) are nearly four times as likely to commit suicide as vets without the progressive neurological disease, according to a recent study. This finding suggests that the Veterans Health Administration (VHA) should increase their suicide prevention efforts among veterans with ALS. The study, “…

Moderate physical activity may slow muscle deterioration associated with the progression of amyotrophic lateral sclerosis (ALS), improving patients’ ability to perform daily life activities, a review study has found. This type of therapeutic exercise also slowed lung function decline, but did not prolong survival, suggesting this form of physical…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

The McQuade Center for Strategic Research and Development (MSRD) and Eikonizo Therapeutics have entered a collaboration to advance potential therapies for rare diseases, including amyotrophic lateral sclerosis (ALS). “We are dedicated to seeking out and supporting novel treatments for complex and rare diseases,” Robert McQuade, PhD, president of MSRD…

To commemorate Feeding Tube Awareness Week, the ALS Association recently highlighted the benefits of feeding tubes in the lives of people with amyotrophic lateral sclerosis (ALS). The association recalled a previous interview with two feeding tube experts at the ALS Center of Excellence at Hennepin Healthcare, in Minneapolis, Minnesota,…