News

Advanz Pharma has acquired the global rights (except in Japan) to market Salagen (pilocarpine hydrochloride) tablets, an approved therapy for dry mouth in people with Sjogren’s syndrome, the company announced. Salagen, originally marketed by Eisai, is also used as a treatment for relieving dry mouth in head…

The Hungarian regulatory authority has agreed to extend the label of Wize Pharma‘s LO2A eye drops to include the treatment of dry eye symptoms in Sjögren’s syndrome patients, the company announced. The treatment is marketed in Hungary as Conheal, by Resdevco. While the formulation is registered and marketed for the treatment of dry…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

Targeting the mTOR signaling pathway may prevent the increased activation and proliferation of immune cells in the salivary glands of patients with primary Sjögren’s syndrome (pSS), according to new research. The study, “Increased mTORC1 activation in salivary gland B cells and T cells from patients with Sjögren’s…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…