The Devil Is in the Details

“Pleased to meet you, hope you guess my name.” — Keith Richards and Mick Jagger, “Sympathy for the Devil” Last week, I experienced an abysmal customer service episode. Lasting the better part of a full workday, the bitter crescendo left…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Lately, I’ve seen an escalation of messages urging me to protect myself from hackers, scammers, and other potential identity thieves. But there’s one identity thief that’s always on my radar: my ALS. That’s because the ongoing symptoms of ALS challenge the very essence of my sense of self. I’ve managed…

Mitsubishi Tanabe Pharma America announced changes to enhance its out-of-pocket assistance program for people with amyotrophic lateral sclerosis (ALS) using Radicava (edaravone). The changes, aimed at making the program easier and more accessible, include a new “hybrid” card meant to help patients obtain appropriate pharmacy and medical co-pay…

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…