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…
“And so castles made of sand Fall into the sea eventually.” — Jimi Hendrix From time to time, given my predilection for quoting songs in my columns, I’m asked whether a song triggers content, or if it’s subject matter in search of lyrics. The answer is, circumstantially, either. On…
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…
Learning I had amyotrophic lateral sclerosis (ALS) was certainly a big adjustment. The changes to my life that slowly chipped away at my goals and sense of self made me feel lost. It was only when I observed ALS patients who were thriving that I was able to appreciate how…
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…
An imaging agent developed by Ashvattha Therapeutics can accurately detect brain inflammation caused by microglia, immune cells that play a key role in neurological diseases such as amyotrophic lateral sclerosis (ALS), a mouse study reported. The company is planning to initiate a Phase 1/2 clinical trial in ALS…
Simultaneous injections of mesenchymal stem cells (MSCs) into the blood and spinal canal were found to delay disease progression in people with amyotrophic lateral sclerosis (ALS) in a small clinical trial. “Our results demonstrated that the IT [intrathecal, or into-the-spine] and IV transplantation of [MSCs] in ALS patients…
In the days after my husband, Todd, was diagnosed with ALS, I was overwhelmed with what lay before us. But Todd went into preparation mode. “We need to sell the house,” he said. “We need to move near your family.” It took more than a year for the house to…
A five-year National Institutes of Health (NIH) grant worth $2.4 million will support an Emory University researcher’s use of artificial intelligence (AI) to accurately and quickly — in real time — decode the electrical signals sent by the brain to control movement. Its ultimate goal is to find ways to “reconnect” the…
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