News

5th NMO Awareness Gala in March Sets $250,000 Fundraising Goal

The Sumaira Foundation (TSF) seeks to raise $250,000 for research into neuromyelitis optica spectrum disorder (NMOSD) and related disorders at its 5th NMO Awareness Gala, set for March 26. The Hollywood-themed event will be held at the Mandarin Oriental hotel in Boston. For ballroom entry, all attendees must…

SB12, Potential Soliris Biosimilar, Fares Well in Phase 1 Trial

SB12, Samsung Bioepis’ biosimilar candidate of Soliris (eculizumab), is comparable to the original medication in terms of safety, pharmacological properties, and immunoreactivity profile, according to data from a Phase 1 clinical trial in healthy volunteers. Soliris, by Alexion Pharmaceuticals, is an approved therapy for a number of conditions,…

Prepare to Light Up Buildings for Rare Disease Day 2022

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

Merck KGaA Buys Chord and Its Oral Cladribine Product for NMOSD

Merck KGaA will continue to advance CRD1 for the treatment of neuromyelitis optica spectrum disorder (NMOSD) and generalized myasthenia gravis (gMG), after the company acquired the therapy’s original developer, Chord Therapeutics. The investigational therapy is an oral cladribine product that works by reducing the number of immune cells, namely…

Uplizna May Be Effective for Patients Without Anti-AQP4 Antibodies

Long-term treatment with Uplizna (inebilizumab-cdon) safely and effectively reduced the frequency of attacks in the 12 neuromyelitis optica spectrum disorder (NMOSD) patients who were negative for antibodies against aquaporin-4 (AQP4) in the N-MOmentum Phase 2/3 trial. Future, appropriately-controlled studies are needed to confirm Uplizna’s benefits in this subgroup…

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.