Amylyx’s experimental oral treatment AMX0035 safely and effectively slows functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to full data from the CENTAUR Phase 2/3 trial. Only changes in upper limb strength were significantly different from those seen in placebo-treated patients, the data show. Meanwhile,…
My husband, Todd, and I celebrated our 17th wedding anniversary in late August with a date at Fitzgerald’s, a restaurant built on the shore of Lake Superior in Eagle River, Michigan. A permit to build on a beach would never be approved today, but it could be done in…
The Phase 3 clinical trial testing an oral suspension formulation of edaravone for amyotrophic lateral sclerosis (ALS) has resumed dosing and patient recruitment, its developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The trial (NCT04165824) was launched in November, but was paused in March due to the COVID-19 pandemic. The study,…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
Looking forward to getting a flu shot is something I never thought possible. But at this point in what has been a very strange year, it’s a darned good excuse to get out and do something. In past years, I followed health guidelines and always waited until early October…
Mutations in the gene C9orf72, a common genetic cause of amyotrophic lateral sclerosis (ALS), cause immune cells to have an abnormally potent inflammatory response when certain receptors are are activated, a new study suggests. The findings could explain why people with ALS are more likely to develop some autoimmune…
The European Commission has granted orphan drug designation to ILB, Tikomed‘s investigational treatment for amyotrophic lateral sclerosis (ALS). In the EU, this designation is based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP), a part of the European…
The U.S. Food and Drug Administration has granted Prosetin orphan drug designation for the treatment of amyotrophic lateral sclerosis (ALS). Orphan drug designation grants financial incentives to companies developing medications for rare diseases, which might not be profitable enough to pursue without government assistance. While a Phase…
In his book “Out of Solitude,” Henri Nouwen writes, “When we honestly ask ourselves which persons in our lives mean the most to us, we often find that it is those who, instead of giving advice, solutions, or cures, have chosen rather to share our pain and touch…
Pharnext’s investigational oral combination therapy PXT864 prevents degeneration of motor neurons and their point of contact with muscle cells, as well as the buildup of toxic TDP-43 protein aggregates — all hallmarks of amyotrophic lateral sclerosis (ALS) — in cellular models of the disease. The data…
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