News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

Processa Pharmaceuticals  announced it has entered into a global licensing agreement with Akashi Therapeutics to possibly develop and market HT-100, Akashi’s candidate to treat diseases associated with tissue fibrosis (scarring) and inflammation, including scleroderma. HT-100 is an experimental, oral small molecule reported to have strong anti-inflammatory…

The U.S. Food and Drug Administration (FDA) has approved Ofev (nintedanib) as the first therapy to slow lung function decline in people with interstitial lung disease (ILD) associated with systemic sclerosis (SSc-ILD), Boehringer Ingelheim announced. SSc affects multiple systems in the body, causing progressive, widespread fibrosis (tissue scarring). Within…

People with systemic scleroderma (SSc) can be prone to problems in the small airways, which can contribute to the cases of lung disease observed in this population, new research found. Airway dysfunction can be detected by impulse oscillometry, a noninvasive and simple…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

EHP-101, an oral cannabidiol treatment candidate for people with scleroderma (SSc) and multiple sclerosis (MS), was found to be safe and well-tolerated in a Phase 1 clinical trial in healthy volunteers. These results clear the way for Phase 2 trials, now in planning stages, to test the effectiveness…

The European Scleroderma Trials and Research Group (EUSTAR) revised activity index scale, known as EUSTAR-AI, is the best tool for predicting the short-term risk of disease progression and severe organ involvement in scleroderma (SSc) patients diagnosed within the last five years, a study reports. It had a better predictive…

Use of vasodilators — medications that trigger the widening and relaxing of blood vessels — may help to prevent heart problems in people with systemic scleroderma, a study suggests. Low-dose aspirin was also found to offer some protection against some forms of cardiac disease, its researchers said, recommending more…

Higher than usual serum levels of uric acid are linked to a greater risk of pulmonary arterial hypertension (PAH) among people with scleroderma, according to new research. Its findings also showed an association between higher levels of this blood biomarker and both disease severity and mortality in scleroderma-associated PAH…