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…
Country music artist Ashley Barron has been named celebrity ambassador of the National Scleroderma Foundation, a role she will take up to increase understanding and awareness of scleroderma as she takes her music on tour this spring and summer throughout the United States. “I am so honored…
UVA1 phototherapy, a form of treatment that uses light for skin disorders, helps ease skin lesions in people with localized scleroderma by regulating certain genes responsible for inflammation and scarring, a study has found. The study, “Clinical and laboratory characterization of patients with localized scleroderma and response to…
Treatment with stem cells grown into a skin-like structure called an organoid reduced inflammation and fibrosis (scarring) in a mouse model of localized scleroderma, a new study found. The study, “Application of an iPSC-Derived Organoid Model for Localized Scleroderma Therapy,” was published in Advanced Science.
A differential blood cell count — a routine test that tells how many cells of each type there are in the blood — may be able to foretell the course of disease in patients with systemic scleroderma (SSc), according to a U.S. study. Higher numbers in both neutrophil counts…
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