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…

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…

Chinese who develop pulmonary arterial hypertension (PAH) from primary Sjogren’s syndrome have worse survival rates if their PAH appears late or if they have poorer cardiac function, a new study shows. The findings, published in the journal Lupus, also show that patients taking immunosuppressants have better survival rates. Sjogren’s syndrome,…

The protocol of a Phase 4 trial testing Wize Pharma‘s LO2A eye drops for the treatment of dry eye symptoms in Sjogren’s syndrome patients has received regulatory approval. Clearance from the Institutional Review Board (IRB) means the study is now allowed to start. The double-blind trial (NCT03319420), to be…

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…

ImmunoQure and Servier have joined efforts to develop a new therapeutic antibody that could benefit patients with Sjögren’s syndrome and systemic lupus erythemathosus. The antibodies will be targeting interferon-alpha (INF-α), a protein needed for immune responses against invading pathogens. Interferon alpha is produced at high levels in…

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…

Pilocarpine is an effective treatment for mouth and eye dryness in patients with Sjogren’s syndrome, a Phase 4 trial from Chile showed. The trial compared the medicine to artificial saliva for 12 weeks in patients with primary and secondary Sjogren’s syndrome. At the end of the study, most patients in the…

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…

A history of infection with nontuberculous mycobacteria — linked to chronic pulmonary infections, but unable to cause tuberculosis — may increase the risk for primary Sjögren’s syndrome, according to a large nationwide Taiwanese study. “Although the exact disease mechanism behind [Sjögren’s syndrome] remains elusive, a variety of environmental, genetic and hormonal…