News

Progressive skin fibrosis within one year increases the risk of lung function decline and mortality in patients with diffuse cutaneous systemic scleroderma (dcSSc), a large-scale observational study found. The finding suggests that skin fibrosis scoring can work as a potential surrogate marker for dcSSc severity in clinical trials and routine…

Systemic sclerosis (SSc) is not associated with clinically significant exocrine pancreatic dysfunction, a condition that can promote malnutrition in patients, researchers report. Of note, exocrine refers to the part of the pancreas that functions as a gland, producing and secreting digestive enzymes. The study, “Exocrine Pancreatic Function is…

Scleroderma patients with ANCA autoantibodies have a higher prevalence of interstitial lung disease (ILD) and pulmonary embolism, as well as a greater mortality risk than those without these antibodies, according to new research. The study, “Significance of anti-neutrophil cytoplasmic antibodies in systemic sclerosis,” was…

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…