To mark what would have been the 79th birthday of the theoretical physicist Stephen Hawking, who lived with amyotrophic lateral sclerosis (ALS) for 55 years, Answer ALS is providing researchers with free access to the world’s largest ALS data portal. To help spread the word, the organization…
Mexiletine, a heart medication used off-label to treat muscle cramps due to amyotrophic lateral sclerosis (ALS), was found to be well-tolerated and to stabilize the “hyperexcitability” of motor neurons in a small group of ALS patients in a pilot Phase 2 trial. Data from the study (NCT02781454)…
“Be nicer,” my daughter whispered to me when I was on the phone telling a restaurant employee my pizza takeout bill was $20 higher than it should be. My tone was apparently sharper than I realized as I went through the math of half off the second pizzas with add-ons…
Imaging data of brain inflammation in people with amyotrophic lateral sclerosis (ALS) that is obtained using different tracers and imaging equipment can be combined using a technique called the “pseudo reference region approach.” This finding may make it easier to conduct multi-center studies about ALS, according to a recent…
The European Medicines Agency has granted orphan drug designation to PrimeC, an investigational combination therapy for amyotrophic lateral sclerosis (ALS). PrimeC, developed by NeuroSense Therapeutics, also has received orphan drug designation in the United States. The designation, both in the U.S. and Europe, encourages drugmakers to develop…
A pivotal clinical trial evaluating pridopidine for the treatment of amyotrophic lateral sclerosis (ALS) has enrolled its first participant, Prilenia Therapeutics, the medicine’s maker, announced. The Phase 2/3 study, dubbed HEALEY ALS Platform – Regimen D Pridopidine (NCT04615923), will compare the safety and effectiveness of…
Mitsubishi Tanabe Pharma America (MTPA) is taking proactive measures to address patient safety in its REFINE-ALS biomarker study during the COVID-19 pandemic. The company announced revisions to the study’s protocol that give participants the option to participate virtually, through the use of telemedicine (medical care delivered virtually) and…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
Nearly half of all patients in a Phase 2 trial of CNM-Au8, Clene Nanomedicine’s lead candidate for the treatment of amyotrophic lateral sclerosis (ALS), showed improvements in the health and functioning of their motor neurons, according to the interim trial data. These data are still blinded,…
“I forgot how dark it is all the time,” my brother commented when he visited us in Michigan’s Keweenaw Peninsula over the holidays. The sun was trapped behind cloud cover for the entire week he was here, and some years, it only peeks out occasionally during January with near daily…
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