$80M Funding Will Help Expansion Develop RNA-targeted Therapies

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…

Cytokinetics is accepting applications from advocacy groups working in cardiovascular and neuromuscular diseases, including amyotrophic lateral sclerosis (ALS), for grants worth $20,000 each that will help them expand their communications and community engagement. In total, five Cytokinetics Communications Fellowship Grants will be awarded winning organizations. The deadline for applications…

The ALS Association has launched a new diagnostic guide — called thinkALS — to aid neurologists in more quickly suspecting and diagnosing amyotrophic lateral sclerosis (ALS) at its early stages. Early diagnosis will allow patients to join clinical trials, and to access treatments and care at disease stages where there’s…

“Does caregiving for ALS ever get easier?” I pondered the question someone had posted on social media. I couldn’t sleep for days after my husband, Todd, was diagnosed. I was consumed with anxiety and fear for the future. How was I going to parent our children as a single mom?…

When I was in elementary school, my mom would occasionally take my brother and me to Woolworth’s, our local five-and-dime store. We would often have lunch there, and my staple was the open-faced roast beef platter, followed by a hot fudge sundae. Afterward, my brother and I were left…

Bionews Insights is launching a survey, in collaboration with ALS News Today, with the goal of understanding how different aspects of this disease affect an individual’s quality of life. The survey, which is expected to take about 15 minutes to complete, opens with WHOQOL-100, a set of questions developed by…

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…

Living with ALS has its challenges. For me, the first year after my ALS diagnosis was the hardest. Looking back, I made some mistakes and wasted time and energy that I wish I could have back again. Although we each experience a different journey with ALS, I believe we all…

Clene, and its wholly owned subsidiary Clene Nanomedicine, have launched an expanded access program (EAP) in the U.S. to allow certain people with amyotrophic lateral sclerosis (ALS) to gain access to its experimental oral therapy CNM-Au8. Called CNMAu8.EAP02, the program will focus on patients who are not eligible…