“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…
Fighting the Predictable Unpredictability of Rare Disease
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…
Columns
What Being Rare Means to Me
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…
Our daughter, Sara, is working on several pieces for an upcoming choir festival. One selection is the song “My New Philosophy” from the musical “You’re a Good Man, Charlie Brown.” The other evening, as our family gathered for dinner, Sara sang the lyrics in which the character Sally tries…
The ALS Canada Research Program and the Brain Canada Foundation partnered to award CA$1.125 million (about $885,600) in grants to advance the understanding, diagnosis, and treatment of amyotrophic lateral sclerosis (ALS). The nine multidisciplinary research grants, awarded late last year to teams across Canada, seek to promote global…
Tau protein levels are unusually high in the motor cortex of amyotrophic lateral sclerosis (ALS) patients with a mutation in the C9orf72 gene, a study reported. The motor cortex, a part of the cerebral cortex responsible for the planning, control, and implementation of voluntary movement, is the brain region most…
Recent Posts
- A dream takes me back to a time before life with ALS
- ALS ONE joining ALS Network to strengthen research, services
- Rollator revamp needed because design matters for living well with ALS
- Study probes high ALS risk in elite athletes and other ‘champions’
- First ALS patient dosed in study of experimental gene therapy VTx-002