Advocates Lobby US Congress During Virtual Rare Disease Week

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

Patient enrollment has begun for Cytokinetics‘ pivotal Phase 3 clinical trial investigating reldesemtiv for the treatment of people with early amyotrophic lateral sclerosis (ALS). The global COURAGE-ALS trial (NCT04944784) will enroll approximately 555 adults who are within two years of their first symptom of muscle weakness.

Cue the heroic Olympic theme song, our competitor is ready to begin! As the timekeeper signals all onlookers to be silent, the athlete draws in a deep breath, then while slowly exhaling, counts out loud from one to 10. She successfully completes the task using just that single breath…

The Paralysis Resource Center (PRC), a comprehensive support program for people with paralysis, was awarded a five-year, multi-million dollar grant by the Administration for Community Living (ACL), part of the U.S. Department of Health and Human Services. Effective as of July, the cooperative agreement between the two runs through June…

The U.S. Food and Drug Administration (FDA) has granted fast track status to Apic Bio’s experimental therapy APB-102, designed for amyotrophic lateral sclerosis (ALS) patients who carry mutations in the SOD1 gene that lead to misfolded proteins in cells. This designation accelerates the development of investigational therapies that…

Mitsubishi Tanabe Pharma America (MTPA) and Target ALS welcome nominations for the Target ALS Rebecca Luker Courage Award, honoring those who have demonstrated courage and made a positive impact on the amyotrophic lateral sclerosis (ALS) community over the past year. “We have dedicated ourselves to a singular…

When my husband, Todd, was diagnosed with ALS, I grieved the loss of our dreams and our future together, but I had no idea how hard daily life would become. ALS caregiving spouses end up taking on a lot of roles. We advocate for resources and battle insurance companies.

BrainStorm Cell Therapeutics announced that a new cleanroom facility for manufacturing NurOwn, its experimental cell-based therapy for amyotrophic lateral sclerosis (ALS), has received good manufacturing practice (GMP) certification from the Israel Ministry of Health. GMPs are standards set to ensure that batches of a medicine are produced with consistent high…

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

“Don’t the best of them bleed it out While the rest of them peter out? Truth or consequence, say it aloud Use that evidence, race it around There goes my hero Watch him as he goes There goes my hero He’s ordinary.” The lyrics of the Foo Fighters’ song…