Mitsubishi Tanabe Pharma Corporation (MTPC) is seeking approval to manufacture and market MT-1186, an oral formulation of edaravone, in Japan for amyotrophic lateral sclerosis (ALS). A similar application recently gained priority review in the U.S., which reduced regulatory review time from the standard 10 months to six months. A…
Mitsubishi Tanabe Seeks Approval of Its Oral Edaravone in Japan
Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…
Treatment with the cell therapy NurOwn may slow the progression of amyotrophic lateral sclerosis (ALS) for those with a specific variation in the UNC13A gene that is linked with a high risk of the disease. Merit Cudkowicz, MD, chief of neurology at the Massachusetts General Hospital, shared the…
Treatment with Clene Nanomedicine’s experimental medicine CNM-Au8 may decrease the risk of disease progression or death in people with early amyotrophic lateral sclerosis (ALS), according to new analyses from the RESCUE-ALS clinical trial. Data also suggest that the therapy helped to prevent the loss of motor neurons in…
I felt twinges of grief as I shopped for a card for a young couple’s wedding. First card: “Wishes on this special day for happiness, joy, and love, along with a future that will bring everything you are dreaming of.” Sigh. I wish my husband and I had a future.
Columns
The Same as It Never Was
“And you may find yourself living in a shotgun shack … And you may ask yourself, ‘Well, how did I get here?’ … Letting the days go by, same as it ever was.” Although I’m not proud of it, my life, pre-ALS, was largely a fortunate stream of autopilot idleness.
An ambitious Irish research project, called Precision ALS, will combine clinical research, data science, and artificial intelligence (AI) to identify the multiple — and potentially targetable — factors involved in the development and progression of amyotrophic lateral sclerosis (ALS). The goal is to use this information to determine which treatments…
If you happened to read my column last week, you’re probably thinking I’m writing this while sitting on a sunny beach, gazing at the ocean, and sipping a tropical drink with a little paper umbrella in it. That’s because I wrote about my busy month filled with ALS-related events,…
Exposure to certain toxic compounds — including beta-N-methylamino-L-alanine (BMAA), formaldehyde, and heavy metals like manganese, mercury, and zinc — increases the likelihood of developing amyotrophic lateral sclerosis (ALS), according to a review paper. Its authors argue that with BMAA — a toxic compound made by algae — enough evidence…
Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
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