News

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

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…

#ECTRIMS2021 – Enspryng Prevents Relapses Over Long Term in Trials

Long-term treatment with Enspryng (satralizumab) safely and sustainably prevents relapses in adolescents and adults with neuromyelitis optica spectrum disorder (NMOSD) who are positive for antibodies against the aquaporin-4 protein (AQP4-IgG), according to four-year data from the SAkura Phase 3 clinical trials. These benefits were observed when the therapy…

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

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.

Uplizna Found Safe at Reducing NMOSD Attacks in African Americans

Treatment with Horizon Therapeutics’ Uplizna (inebilizumab-cdon) was found safe and effective at reducing the frequency of attacks in African-American adults with neuromyelitis optica spectrum disorder (NMOSD), according to a retrospective analysis of the N-MOmentum clinical trial. These findings were presented in a poster at the 15th World Congress…

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

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…

COVID-19 Vaccines Well-Tolerated by NMOSD Patients, Survey Finds

COVID-19 vaccines are safe and well-tolerated by people with the rare neurological disorders neuromyelitis optica spectrum disorder (NMOSD), MOG-antibody disease (MOGAD), and transverse myelitis in the U.S., according to a recent study. Patients surveyed about their vaccine experiences reported a rate of side effects similar to that of the…

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…