A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
News
MND-SMART, a pivotal platform trial designed to simultaneously test multiple treatment candidates in people with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases, has opened its third clinical site in Scotland. This type of platform trial, distinct from typical clinical trials that test a single treatment…
A $2 million grant from the National Institutes of Health (NIH) is funding work to better understand how molecular structures called protein-RNA condensates form and are regulated within cells. Findings from the five-year project could help in understanding and treating diseases where these molecular structures play a role, including…
Head trauma, electrical burns, and hobbies involving the toxic metal lead appear to be risk factors for developing amyotrophic lateral sclerosis (ALS), a U.S.-based study found. Holding a job in mechanics, painting, or construction also significantly increase the chances of developing the disease, researchers noted. The study, “…
A metabolite of testosterone, dihydrotestosterone (DHT), is found at significantly lower levels in the cerebrospinal fluid of people with amyotrophic lateral sclerosis (ALS), a small study found. This metabolite, or hormone, is likely critical for the survival of motor neurons, and its lack in the central…
Treatment with AMX0035, Amylyx’s experimental oral therapy, significantly extends the lives of amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to a long-term survival analysis of the CENTAUR Phase 2/3 trial. The new data were reported in a study titled “Long‐Term…
October is International Augmentative and Alternative Communication (AAC) Awareness Month, set aside annually to inform the public about how amyotrophic lateral sclerosis (ALS) patients and others use these devices to communicate. From educational presentations to personal videos, supporters globally are marking the event organized by the International…
With a €1 million (about $1.1 million) prize being offered, researchers are invited to join a challenge that seeks to give people with motor neuron diseases (MND) – including those with amyotrophic lateral sclerosis (ALS) – improved independence and a more normal lifestyle. The Cullen Education and…
Most people with bulbar onset amyotrophic lateral sclerosis (ALS) showed distinct signs of motor neuron injury starting in the bulbar region of the brain that controls swallowing and speaking, before symptoms descended to regions that control the upper limbs and then the lower…
A Phase 2 and potentially pivotal study investigating pegcetacoplan (APL-2) as a potential treatment for amyotrophic lateral sclerosis (ALS) is now enrolling patients, Apellis Pharmaceuticals, the therapy’s developer, announced in a press release. The MERIDIAN trial (NCT04579666) is enrolling up to 228 adults with sporadic ALS and…
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