Kadmon Holdings started a Phase 2 clinical trial testing its inhibitor KD025, with the first patient with diffuse cutaneous systemic sclerosis (SSc) being dosed, the company announced. KD025 is a selective, orally available inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), an enzyme involved in signals that modulate the…
Being older, male, using opioids, having the diffuse cutaneous disease type, and muscle disease, increase the risk of pseudo-obstruction in people with systemic sclerosis (SSc), a study shows. The research, “Evaluation of risk factors for pseudo-obstruction in systemic sclerosis,” was published in the journal…
Different types of systemic sclerosis-related autoantibodies may lead to different presentations of the disease, and affect distinct groups of internal organs, research suggests. The study reporting the findings, “Clinical Manifestation and Incidence of Cardiopulmonary Complications in Early Systemic Sclerosis Patients with Different Antibody Profiles,” was published…
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
High levels of two proteins in the blood, called KL-6 and CCL-18, may work as biomarkers for predicting disease severity and progression of interstitial lung disease (ILD) in systemic sclerosis (SSc) patients, Phase 2 trial data suggest. The study, “Pneumoproteins KL-6 and CCL-18 Predict Progression of Interstitial Lung Disease…
Older age, active digital ulcers, lung tissue scarring, muscle weakness, and increased levels of C-reactive protein (CRP) are predictive factors of progressive organ damage in people with systemic sclerosis (SSc), a study has found. Taking these factors into consideration may help clinicians to identify patients at risk of SSc worsening,…
Tagraxofusp, a medicine approved in the U.S. under the name Elzonris to treat a rare blood cancer, may help decrease levels of immune cells that promote inflammation in systemic sclerosis (SSc), new preclinical data suggest. The results were shared in a poster, “CD123+ plasmacytoid dendritic cells (pDCs) from…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Injections of hyaluronidase, marketed as Hylenex, among other names, improved the ability to eat and reduced difficulties in opening the mouth and dental care in a woman with scleroderma, according to a new case report. The case was described in a study, “Hyaluronidase Treatment of…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
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