News

When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress — SMARD, for short. Hunter Pageau, a 12-year-old boy from North Haven, Connecticut, is one of only 80 people in the world known to have SMARD, a motor neuron disease…

Sjogren’s syndrome patients with chronic dry eyes have higher medical costs than people with dry eyes who don’t have Sjogren’s, a study from Eastern China shows. The research also points to a link between anxiety and medical costs for dry eye treatment in Sjogren’s patients. The study, “…

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…