A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
Chemomab Therapeutics is teaming up with the University of Leeds to investigate the role of the protein CCL24 in scleroderma (SSc) — particularly how it contributes to blood vessel damage. Their findings may aid in the clinical development of CM-101, Chemomab’s first-in-class antibody designed to target and…
Having pulmonary hypertension shortens survival in people with scleroderma, also called systemic sclerosis (SSc), according to a Brazilian study that followed patients for up to three years and used a computer algorithm to analyze the results. Specifically, this lower survival rate in SSc patients with pulmonary hypertension was…
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
The delayed passage of food waste through the colon — or slow colonic transit — is common and often severe in people with scleroderma, a study has revealed. This condition was more likely in patients with widened blood vessels seen on the skin and less likely in those with…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
Altered levels of immune signaling proteins (cytokines) and fat-based hormones in systemic sclerosis (SSc) are associated with changes in body mass, a blood analysis determined. Researchers say their findings suggest that “an abnormal twist … takes place in SSc” between the immune signaling proteins, these hormones, and body mass index or BMI,…
A new study being conducted at the University of Pittsburgh is testing the safety and effectiveness of COVID-19 vaccines in people with scleroderma and other autoimmune diseases. The CoVER study seeks to enroll adults, 18 or older, who have rheumatoid arthritis, Sjogren’s syndrome, myositis, or…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
Sanofi has entered into a merger agreement with Kadmon, which is developing belumosudil (KD025), an oral investigational treatment for people with the diffuse cutaneous form of systemic sclerosis (dcSSc). Belumosudil, sold under the brand name Rezurock, was recently approved for patients, 12 years and older, with chronic…
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