Patricia Inácio, PhD, science writer —

An investigational RNA molecule called an RNA aptamer halted the death of motor neurons and helped preserve motor function in a mouse model of amyotrophic lateral sclerosis (ALS). Further research is ongoing to confirm the findings in additional ALS models before testing it in humans, “but I’m optimistic that this…

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Spinogenix’s investigational oral molecule SPG302 for treating amyotrophic lateral sclerosis (ALS). Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer than 200,000 people in the…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…

Clearance was given to open a Phase 1/2 trial evaluating APB-102, a potential gene therapy for amyotrophic lateral sclerosis (ALS) patients who carry mutations in the SOD1 gene, its developer Apic Bio announced. The trial is scheduled to begin later this year or early next year, following U.S. Food and Drug Administration…

A global Phase 3 trial of AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS)  intends to enroll up to 600 patients, the therapy’s developer, Amylyx Pharmaceuticals, announced in a press release. Likely to begin in the coming months, the trial — to be called PHOENIX…

A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The Motor Neurone Disease (MND) Association and Toyota (GB), the U.K. division of Toyota, have established a partnership to raise funds and improve awareness for the needs of people with disorders that include amyotrophic lateral sclerosis (ALS). The staff at Toyota (GB) selected MND Association as the company’s…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Actimed Therapeutics is developing S-oxprenolol to prevent muscle wasting and body mass loss due to amyotrophic lateral sclerosis (ALS), using proceeds from a recent license agreement with Faraday Pharmaceuticals. Under the agreement, Faraday acquired global rights to develop and commercialize S-oxprenolol for muscle-affecting disorders other than ALS, particularly…