Emerald Health Pharmaceuticals’ cannabis-derived therapy, EHP-101, eased the symptoms of systemic sclerosis by preventing blood vessel damage, and reducing fibrosis and inflammation in a study using mice. The study, “Cannabinoid derivatives acting as dual PPARγ/CB2 agonists as therapeutic agents for Systemic Sclerosis,” was published in…
Boehringer Ingelheim has submitted its proprietary therapy nintedanib to the U.S. Food and Drug Administration and the European Medicines Agency for regulatory approval to treat systemic sclerosis (SSc)-associated interstitial lung disease (SSc-ILD). Approval is needed by the relevant regulatory agencies before a new product or treatment can reach…
A high prevalence of pulmonary veno-occlusive disease (PVOD) in systemic sclerosis (SSc) patients with pulmonary hypertension associated with interstitial lung disease may explain the poor prognosis observed in this patient group, according to a new study. The study, “Pulmonary veno-occlusive disease is highly prevalent in scleroderma patients undergoing…
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 Bathdissertation, she analyzed Brexit’s long-term impact…
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,…
The first patient with diffuse cutaneous systemic sclerosis (SSc) has been given the investigational therapy AVID200 in an ongoing Phase 1b trial, announced the therapy’s developer Forbius. The open-label study (NCT03831438), which is currently recruiting participants, was designed to assess the safety and tolerability of AVID200, as well…
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…
Patients with systemic sclerosis (SSc) have reduced health-related quality of life compared with other systemic rheumatoid diseases, a new study shows. A significant association between skin thickness scores and impaired quality of life also was reported in the study. The study “Health-related quality of life in systemic sclerosis…
There was no significant difference between placebo and lanifibranor, Inventiva Pharmaceuticals’ investigational treatment for systemic sclerosis, in alleviating skin fibrosis in a Phase 2b study, the company announced. Based on the results, Inventiva will no longer proceed with the clinical development of lanifibranor for scleroderma. It will…
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…
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