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…
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…
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…
“What you need, my son Is a holiday in Cambodia Where people dress in black.” Those lyrics from the song “Holiday in Cambodia” by the punk rock band Dead Kennedys illustrate how counterintuitive any public holiday display may appear. Without knowing the context, all-black attire may seem odd. Yet,…
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…
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…
“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…
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…
