Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…
News
A detailed atlas of the various types of cells that populate the motor cortex, the brain region that controls voluntary movement and is damaged in people with amyotrophic lateral sclerosis (ALS), was created by a worldwide consortium of researchers. The long-term goal of the group, which involves hundreds of…
Tofersen, an experimental treatment for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, failed to significantly slow the rate of disease progression relative to a placebo in a Phase 3 trial. These top-line findings mean that the VALOR study (NCT02623699), which is testing tofersen in…
The ALS Association is reminding amyotrophic lateral sclerosis (ALS) patients and caregivers that the open enrollment period for Medicare recipients to make changes to their existing coverage runs through Dec. 7. The open enrollment period for altering coverage opened Friday. After Dec. 7. changes will be allowed only after…
Note: This story was updated Oct. 19, 2021, to note that the trial is taking place at 90 clinical sites and to update the contact information, based on additional information from the company. A Phase 3b clinical trial is recruiting adults with amyotrophic lateral sclerosis (ALS) to assess the…
Amgen and Neumora Therapeutics announced that they are collaborating to develop precision therapies for amyotrophic lateral sclerosis (ALS) and other brain diseases. The partnership will leverage each of the company’s expertise, with the goal of bringing to the market effective therapies targeted at those patients most likely…
A signaling protein called neurturin promotes the development of muscles with greater endurance, leading to better exercise performance and coordination, according to a new mouse study. The results suggest that therapeutic approaches using neurturin — for example, using a gene therapy that encodes the protein — might be useful…
The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…
An investigational treatment for amyotrophic lateral sclerosis (ALS), SBT-272 was found to sustainably reach different brain regions, and to protect mitochondria — a cell’s energy source — from TDP-43 toxic aggregates in a mouse model of the disease. “We are excited about the promise of SBT-272 as a potential therapeutic…
Early trials in healthy adults supported the safety and tolerability of two potential oral therapies for amyotrophic lateral sclerosis (ALS) — DNL343 to prevent the cellular stress that can promote toxic protein granules, and DNL788 to block a protein linked to brain inflammation — Denali Therapeutics reported. A Phase…
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