News

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

Ketamine Earns FDA’s Orphan Drug Designation

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…

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…

Enrollment Open for COURAGE-ALS Trial of Reldesemtiv

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.

Grants Support Reeve Foundation’s Paralysis Resource Center

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…

FDA Puts APB-102 Gene Therapy for SOD1 ALS on Fast Track

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…