News

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…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

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…

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…