News

Sumaira Foundation Awards Five $25K Grants to Advance Research

The Sumaira Foundation (TSF) has awarded five $25,000 grants to projects seeking to advance research into preventing, treating, and potentially curing neuromyelitis optica spectrum disorder (NMOSD). This year’s awards — four SPARK grants and one Unicorn grant — will help researchers initiate projects focused on: biomarkers…

Rare Disease Groups in US Join in Plea for Care Across State Lines

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…

Analysis: Uplizna Provides Long-term NMOSD Attack Prevention

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.

Global Genes, Diversity Coalition Team Up to Advance Health Equity

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…

#ECTRIMS2021 – Greater B-cell Depletion With Uplizna Linked to Better Outcomes

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…

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…