Amylyx’s AMX0035 Now Under Review for Approval in Europe

The European Medicines Agency (EMA) has agreed to review a marketing authorization application (MAA) from Amylyx Pharmaceuticals requesting the approval of AMX0035 — a combination of two compounds — for the treatment of people with amyotrophic lateral sclerosis (ALS). The validation comes nearly two months after Amylyx filed the…

A research team in Canada received a CA$1.6 million (almost $1.25 million) grant from the Weston Family Foundation to study the benefits of a specific probiotic in people with amyotrophic lateral sclerosis (ALS). Probiotics — found in yogurt, fermented foods, and dietary supplements — are live microorganisms thought to improve…

I set my husband, Todd, up on his computer with his HeadMouse and sip-and-puff clicker. ALS has compromised his breathing, so I put on his noninvasive ventilator. I call my mom, who lives next door. She will keep her mobile phone in her pocket. If Todd needs anything, he…

Health Canada has authorized a new drug submission for masitinib — AB Science’s add-on therapy for amyotrophic lateral sclerosis (ALS) — under the notice of compliance with conditions policy (NOC/c), designed to get new medications to patients more quickly. If granted, this would allow conditional marketing of…

The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…

“Will you still need me, will you still feed me, When I’m sixty-four” Paul McCartney wrote the song “When I’m Sixty-Four” four decades before reaching that age himself. McCartney admitted that the number was chosen arbitrarily. He later quipped during an interview that 65 would have been a…

In response to the National Institute of Neurological Disorders and Stroke‘s (NINDS) request for community input regarding what it should prioritize to accelerate amyotrophic lateral sclerosis (ALS) research, the ALS Association’s overarching message is this: move swiftly to help patients. NINDS, which is part of the National…

Throughout February, many patient columnists like me who write for Bionews, the parent company of this website, are writing columns recognizing Rare Disease Month, which culminates in Rare Disease Day on Feb. 28. Not only are we encouraging our readers to reach across patient community lines to learn…

Aquinnah Pharmaceuticals has partnered with Roche to further develop its brain-penetrating, oral small molecules for the treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. These small molecules are designed to eliminate the persistent stress granules that contribute to the buildup of toxic protein clumps in…

It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.