The U.S. Food and Drug Administration (FDA) has accepted and granted priority review to an application for Esbriet (pirfenidone) as a treatment for unclassifiable interstitial lung disease (UILD). The supplemental new drug application was submitted by Genentech, Esbriet’s developer and a member of the Roche Group, based on…
Galapagos NV and Gilead Sciences are discontinuing all clinical trials with the investigational medication ziritaxestat. This includes the long-term extension of the Phase 2a NOVESA trial in scleroderma, as well as the Phase 3 ISABELA program in idiopathic pulmonary fibrosis (IPF). The decision is based on…
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
Autoantibodies against proteins of the telomeres — the protective caps of chromosomes and a marker of lifespan — were found in scleroderma patients with lung disease and shorter telomeres, a new study reveals. The findings suggest these autoantibodies could serve as a novel biomarker for scleroderma with lung disease.
In a study of the entire working population of Denmark, exposure to respirable crystalline silica — common in jobs in the construction industry — was linked to an increased risk of developing autoimmune rheumatic disorders, including scleroderma and rheumatoid arthritis. The dose-dependent nature of the association,…
While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…
A tiny RNA molecule called microRNA-320a is associated with regulating collagen levels and fibrosis, or scarring, in systemic sclerosis (SSc) complicated by interstitial lung disease (ILD), a study reports. According to investigators, this is the “first…
Small genetic variations that alter gene activity could play a role in the development of scleroderma and open new avenues for treating the disorder, a study indicates. Many of the genes identified through this work also show unusual levels of activity in tissues often affected in this disease. The study,…
Intensified B-cell depletion therapy (IBCDT) — administering immunosuppressants to lower the number of antibody-producing immune B-cells — may be a promising strategy to help people with scleroderma manage their condition, a study shows. IBCDT may be particularly helpful for those with extensive skin involvement and severe…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…
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