While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…
The Phase 2 clinical trial investigating Clene Nanomedicine‘s CNM-Au8 as a potentially disease-modifying therapy for people with amyotrophic lateral sclerosis (ALS) has enrolled more than half of its participants ahead of schedule. RESCUE-ALS (NCT04098406) dosed its first participant in January,…
On Memorial Day, my mom, the kids, and I visited Lakeside Cemetery in Hancock, Michigan. My mom and the kids left in time to attend the Memorial Day service. I arrived late, just as it concluded, because I needed to help Todd in the bathroom and then…
Mitsubishi Tanabe Pharma America (MTPA) has set up a support program for adults living with amyotrophic lateral sclerosis (ALS) and wanting to learn more about this disease and Radicava (edaravone), an intravenous ALS treatment the company markets in the U.S. Called JourneyMate, the program connects ALS patients…
A large part of successfully living with ALS is to face each challenge with a positive mindset and find workable solutions so I can continue to move forward with my life. Lately, a 4-by-6-inch piece of cloth — yes, a face mask — is putting me to the test. What’s…
The U.S. Food and Drug Administration (FDA) has given fast track designation to Orphazyme’s investigational therapy arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). A therapy candidate is put on the FDA’s fast…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Levels of creatinine kinase — a marker of muscle damage — are elevated in most amyotrophic lateral sclerosis (ALS) patients and appear to be significantly higher among those whose disease progresses at a slower pace, a study in patients and mice shows. These findings suggest that this protein, whose rise…
A few years before my husband, Todd, was diagnosed with ALS, he baked a cake for a church fundraiser with one hand while our infant daughter slept on her stomach across his other forearm, her head cradled in his hand. It was impressive enough that he could make the traditional…
Changing the composition of bacteria in the gut using antibiotics or fecal transplants may help prevent or reduce the severity of symptoms of amyotrophic lateral sclerosis (ALS), a study in mice with the most common ALS mutation shows. This gut-brain connection may help explain why some patients with this…
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