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.
A U.S. Food and Drug Administration (FDA) advisory committee has scheduled a virtual meeting with Amylyx Pharmaceuticals to review its application for the approval of AMX0035 to treat amyotrophic lateral sclerosis (ALS), the company announced. The online meeting to discuss data supporting the approval request is set for March 30.
The investigational antibody therapy for amyotrophic lateral sclerosis (ALS), AT-1501, will now be called tegoprubart, its maker, Eledon Pharmaceuticals, has announced. The change comes after the United States Adopted Names (USAN) Council selected tegoprubart as the unique generic, or nonproprietary, name for the therapy candidate. The company…
