News

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

Therapy with anti-tumor necrosis factor (anti-TNF) is highly effective in people with ankylosing spondylitis. But researchers suggest that patients whose treatment lost its effectiveness or resulted in adverse events can benefit from switching anti-TNF therapies. These are the conclusions of a review study, “…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

Results from a Phase 3 clinical trial show that treatment with Cosentyx (secukinumab) led to fast, significant, and sustained symptom improvement in patients with active ankylosing spondylitis (AS). Improvements were particularly noticeable in patients who had never been treated with anti-TNF (tumor necrosis factor) therapies. The study, “…

Treatment with the monoclonal antibody Taltz (ixekizumab) significantly reduced the symptoms of radiographic axial spondyloarthritis (rad-axSpA) in patients participating in a Phase 3 clinical trial, early data show. The study, called COAST-V (NCT02696785), met its primary and all key secondary endpoints, therapy developer Eli Lilly said in a…

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

Active inflammation in the bone is a major risk factor for bone loss in patients with ankylosing spondylitis. That finding was in the study “Bone edema on magnetic resonance imaging is highly associated with low bone mineral density in patients with ankylosing spondylitis,” which was published in the open-access…

In recognition of Rare Disease Day 2018, BioNews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

Treatment with Merck’s Simponi (golimumab) is now recommended for patients with severe non-radiographic axial spondyloarthritis (nr-axial SpA) in England and Wales. Nr-axial SpA and ankylosing spondylitis (AS) are collectively called axial spondyloarthritis. The term Nr-axial SpA was originally used to define cases of AS that do not exhibit radiographic evidence…