The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
Taking part in one Olympic event is an extreme accomplishment — but a U.K. couple are engaged in their version of 96 Olympic events, from synchronized swimming and skateboarding to cycling and wrestling, all in an effort to raise £30,000 (about $41,620) to support the Motor Neurone Disease Association…
Thanks to a new partnership, every amyotrophic lateral sclerosis (ALS) patient in New Jersey, about 400 in total, will have free access to eye gaze technology that can aid them in communicating. As the name suggests, this assistive technology helps people with neurodegenerative diseases, such as ALS, to communicate…
A £500,000 (almost $700,000) grant the My Name’5 Doddie Foundation was given to the LifeArc & MND Association Translational Research Fund to support work that might lead to new treatments for motor neuron diseases (MND), including amyotrophic lateral sclerosis (ALS). The fund, jointly established by LifeArc…
Amylyx Pharmaceuticals has raised $135 million to advance the clinical development and the potential launch of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). The Series C financing round, led by Viking Global Investors, also will be used to support further research and…
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…
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