Diminished activity in the C9orf72 gene that is linked with amyotrophic lateral sclerosis (ALS) affected the neuromuscular junction — where nerve and muscle cells connect — in a zebrafish model, establishing the model as a disease research tool and the gene’s role in ALS symptoms. Zebrafish in this C9orf72 loss-of-function model…
Men who played professional soccer were nearly four times more likely to develop a neurodegenerative disorder, including a motor neuron disease such as amyotrophic lateral sclerosis (ALS), than men of similar age in the general population, a long-term analysis found. Risk was greatest in players with longer careers and…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ketamine, PharmaTher’s investigational therapy for amyotrophic lateral sclerosis (ALS). Orphan drug status is intended to encourage the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. It provides…
Actemra (tocilizumab), an approved treatment for rheumatoid arthritis, was found to be safe and well-tolerated, and to reduce a key marker of inflammation in amyotrophic lateral sclerosis (ALS) patients with evidence of systemic inflammation in a Phase 2 study. Trial results did not demonstrate that Actemra could slow ALS disability…
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.
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…
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…
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