According to a story from Scleroderma News, findings from the recent Phase 2 EDITA clinical trial suggested that ambrisentan (marketed as Letairis), which is approved to treat pulmonary arterial hypertension…
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…
Right now, no cure exists for people living with systemic sclerosis (also known as systemic scleroderma). There are treatment options available to manage systems. However, these also come with issues.…
A study led by a team of German scientists provides evidence of the safety and efficacy of CD19 CAR T cell therapy and its impact on autoimmune diseases. The study…
Although the medical field has made significant strides over the years, there is still a lot we don’t know about the body. For example, what can we learn about…
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…
Initially, belimumab (sold under the brand name BENLYSTA) was approved for the treatment of patients for lupus and lupus nephritis. It was the first FDA-approved biologic treatment within this indication.…
According to a story from St. George News, when painter Linda Toombs was diagnosed with systemic scleroderma, a rare autoimmune disease, doctors told her that she wouldn't see her children…
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…
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