For over thirty years, researchers at the University of Pittsburgh (UPMC) have been observing cases of limited scleroderma, a connective tissue disorder that causes thick hard patches of skin. Scleroderma…
Earlier this month, I wrote about how efzitimod, a therapy for systemic sclerosis, received Orphan Drug designation from the FDA. Now, another therapy – this one from Gesynta Pharma (“Gesynta”)…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, “rare”…
Prometheus Biosciences Inc. has just announced positive results from their Phase 1 investigation for a new treatment for systemic sclerosis-associated interstitial lung disease (SSC-ILD). This trial investigated PRA023, an anti-TL1A…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
While there is no cure for patients with systemic scleroderma, there are some therapies to manage symptoms, such as medications, antibiotics, physical therapy, or even surgery. But according to Medical…
In an article published in The American Journal of Managed Care (AJMC), researchers provided an overview of the current treatment and management paradigm for systemic-sclerosis (also called scleroderma) with interstitial lung…
It can take years for patients with rare diseases to get diagnoses and understand more about their conditions. But what if someone developed a new and efficient technology that could…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Pulmonary fibrosis is a condition that is caused by scarring of the lungs which can lead to shortness of breath and other respiratory issues. Some cases of COVID-19 have lead to…
Recently, biopharmaceutical company Acceleron Pharma Inc. ("Acceleron") announced that its investigational therapy ACE-1334 was granted Orphan Drug designation by the FDA. The treatment is designed for patients with systemic…
According to a story from PR Newswire, the drug company CiVi Biopharma, Inc. today announced that its subsidiary Eicos Sciences, Inc. has resumed recruiting patients for its phase 3 clinical…
By Lauren Thayer from In The Cloud Copy Systemic sclerosis is a rare autoimmune disease that is characterized by vascular abnormalities and fibrosis of various parts of the body, including…
Biotechnology company Talaris Therapeutics, Inc. submitted an Investigational New Drug (IND) application to the FDA for their novel cell therapy treatment FCR001. As per a press release, the FDA…
According to a story from Scleroderma News, June is recognized as Scleroderma Awareness Month. While physical distancing as a result of the ongoing coronavirus/COVID-19 pandemic may keep some folks at…
Every day, we learn more and more about COVID-19. This viral pandemic has nearly 4.9 million diagnosed cases worldwide, with 1.5 million in the United States alone. As more about…
The understanding of rare diseases is often lacking, making it difficult for rare disease patients to get the diagnosis and treatment that they need. Dr. Tracy M. French aims to…
by Danielle Bradshaw from In The Cloud Copy A year-long study has shown that Tracleer greatly reduces the skin-related symptoms of scleroderma. The results also showed that although it helped…
A news release from Benzinga shares that biotechnology company Emerald Health Pharmaceuticals is beginning a Phase 2 clinical study on EHP-101 as a treatment for patients with systemic sclerosis.…
According to a story from Pulmonary Hypertension News, the results of a recent phase 3 clinical trial have determined that patients with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) stand…
CSL Behring has recently received the Orphan Drug designation for Privigen for the treatment of systemic scleroderma. Privigen has also been granted the Fast Track designation. This therapy has been…
By Jodee Redmond from In The Cloud Copy A recent study suggests that certain antibodies could be linked to specific human leukocyte antigen (HLA) genes and environmental components in scleroderma.…
According to a story from Benzinga, the biotherapeutics company CSL Behring has recently announced that its medical product PRIVIGEN ® has earned Orphan Drug designation from the US Food and…
According to a story from Pulmonary Hypertension News, a recent study has determined that cardiopulmonary exercise testing can be an effective method to help identify systemic sclerosis patients that are…
It is already known that gender plays a role in systemic sclerosis disease with scleroderma – it’s about four times as prevalent in women than men - but a…
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