Dosing has begun in a clinical trial of WVE-004, Wave Life Sciences‘ investigational therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with C9orf72 gene mutations. “ALS and FTD are devastating illnesses where therapeutic progress has been extremely limited. Advancing discovery and development of new treatments…
A subsidiary of Sperogenix Therapeutics has acquired exclusive rights to develop and commercialize ABSK021, an investigational oral therapy for amyotrophic lateral sclerosis (ALS) and other rare neurological diseases in mainland China, Hong Kong, and Macao. Under the terms of the agreement between Sperogenix MedTech and Abbisko Therapeutics, the therapy’s…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
Treatment with the investigational oral therapy masitinib can extend lifespan when given early in the course of amyotrophic lateral sclerosis (ALS), according to a new study. “These long-term survival data, with an average follow-up of 75 months since diagnosis, suggest that masitinib can offer a substantial survival benefit…
AB Science’s proposed measures to ensure patient safety in clinical trials of its experimental oral therapy masitinib were deemed sufficient by the French National Agency for Medicines and Health Products Safety (ANSM), and patient enrollment in the country may resume. The announcement comes less than two months after…
In keeping with its commitment to turn amyotrophic lateral sclerosis (ALS) into a “livable disease” by 2030, and to ramp up discovery and funding of new therapy candidates, the ALS Association is moving to a unified structure from a federated one. “Our best opportunity to fulfill this promise and…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
I’ve been thinking about time travel after watching a couple movies with my husband, Todd, who has ALS. We enjoyed “About Time” during one of our date nights and “The Time Traveler’s Wife” during another. Both movies have subplots that involve love, marriage, and children. If time travel…
Verge Genomics and Eli Lilly have entered into a three-year collaboration to find and develop therapies for amyotrophic lateral sclerosis (ALS). Verge will focus on discovering and validating potential new therapeutic targets, while Lilly will select up to four candidates to advance through clinical testing with a goal of…
“So, round and around and around we go, Where the world’s headed, nobody knows. Oh, great googa-mooga, can’t you hear me talking to you? Just a ball of confusion, Oh yeah, that’s what the world is today.” The Temptations song “Ball of Confusion” was released in 1970, following the…
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