News

Uplizna (inebilizumab-cdon) safely prevented autoimmune attacks in 83% of people with neuromyelitis optica spectrum disorder (NMOSD) who received the treatment for four or more years, data from the N-MOmentum Phase 2/3 clinical trial show. Among those who experienced relapses, most did so during the first year of treatment.

Flying can be an ordeal for many people with disabilities who rely on wheelchairs to get from point A to point B. Sometimes disabled travelers get to their destination only to realize their wheelchair or scooter is broken or missing. In 2019, the year after airlines were required to release…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Treatment with Horizon Therapeutics’ Uplizna (inebilizumab) leads to a rapid and sustained drop in immune B-cells in adults with neuromyelitis optica spectrum disorder (NMOSD), data from the N-MOmentum Phase 2/3 clinical trial show. Notably, while all Uplizna-treated patients experienced significant clinical benefits, those with greater B-cell depletion showed lower…

Note: This story was updated Nov. 2, 2021, to note that Soliris is taken every two weeks, rather than weekly. Soliris (eculizumab) is more effective than Enspryng (satralizumab) and Uplizna (inebilizumab) at lowering the risk of relapse in adults with neuromyelitis optica spectrum disorder (NMOSD) who are…

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…

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…

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.

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…

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…