News

People with systemic scleroderma (SSc) who have certain autoantibodies are at increased risk of developing pulmonary hypertension, or high pressure in the blood vessels of the lungs, according to a study in the U.S. Researchers also observed that patients who tested positive for these autoantibodies, called anti-Th/To antibodies,…

Among people with systemic scleroderma (SSc), those who have digital ulcers (DUs) — sores on their fingers or toes — tend to use more healthcare resources, a new study suggests. “Our study demonstrates increased health resource utilization by SSc patients with DUs compared to those without DUs in a…

Local injection with botulinum toxin A (BTX-A) safely and effectively reduces Raynaud’s phenomenon (RP) and nailfold small blood vessel, or capillary, abnormalities in women with scleroderma, according to data from a small study in China. Treatment also led to significant improvements in upper limb function in women with…

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Gesynta Pharma’s GS-248, its oral therapy for people with systemic sclerosis (scleroderma). Orphan drug status aims to encourage therapies for rare diseases through benefits such as seven years of market exclusivity and exemption from FDA fees. “The…

A protein that triggers a form of cell death driven by inflammation is present at high levels in the skin of patients with scleroderma, and this suggests it may play a role in the development of the disease, according to a new study. Researchers also observed that the skin…

Fat-derived cells from adults with diffuse cutaneous scleroderma that are then injected under the skin of the hands led to clinically meaningful improvements in hand function, according to published data from the Phase 3 STAR I clinical trial. The safety and efficacy of using these cells, called adipose-derived regenerative…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to efzofitimod (ATYR1923) to treat interstitial lung disease (ILD) in people with systemic sclerosis (SSc). The therapy was developed by aTyr Pharma for treating severe diseases affecting the lungs. Efzofitimod was designated as an orphan drug…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…