More Studies Confirm Hefty Economic Burden of Rare Diseases

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…

Since February is known as the month of love, I’d like to shower some love on all the ALS caregivers around the world. Add a special shoutout to family caregivers — the spouses, partners, family members, friends, and even neighbors who care for someone with ALS. It’s a challenging responsibility…

Although not thought common, laryngospasm — the sudden contraction of the vocal cords that makes speaking and breathing temporarily difficult — does affect some with amyotrophic lateral sclerosis (ALS), a study reported. Excess saliva irritating the vocal cords was the most notable trigger for laryngospasm in their work, the researchers…

Mutations in the FUS gene, which cause some forms of amyotrophic lateral sclerosis (ALS), result in a circular RNA molecule called circ-Hdgfrp3 being pulled into toxic protein clumps that form in nerve cells, according to a study. This is the first time that a circular RNA has been shown…

The Cullen Education and Research Fund (CERF), a private philanthropy based in London, has awarded more than $800,000 to support two U.S. research projects that are working to improve life for people with amyotrophic lateral sclerosis (ALS). The CERF Medical Engineering Prize, worth €500,000 (about $570,000), was awarded to…