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…
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…
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…
Dimethyl fumarate or DMF — an oral therapy approved for multiple sclerosis (MS) — was well-tolerated among adults with amyotrophic lateral sclerosis (ALS) but failed to significantly slow disease progression or to improve survival and quality of life in these patients, according to data from a Phase 2…
A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.
Expansion Therapeutics has raised $80 million in funding to support the development of its small molecule RNA platform, called SMiRNA, for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The Series B financing will help the drug development company use its platform to discover small molecules targeting RNA…
Research into gene therapies for motor neuron diseases (MNDs), a group of progressive neurological disorders that includes amyotrophic lateral sclerosis, is set to begin at the University of Sheffield in collaboration with Cell and Gene Therapy Catapult. Supported by a £513,141 (about $700,00) grant from LifeArc and the…
