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…
Studies show that there is a relationship between illness or death (morbidity or mortality), scleroderma renal crisis (SRC), and a person’s race. A study using data from the U.S. Military…
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…
When Taylor Brown was just two years old, her mother TaMetress Reed began noticing certain symptoms, such as hardening and tightening patches of skin. As Brown grew up, the symptoms…
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…
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 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…
This year, because of COVID-19, many healthcare-related events and symposiums have been moved online. However, that doesn't mean that the insights are any less helpful! In the virtual ACR…
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…
According to a recent press release from XBiotech, a Texas-based biotechnology company, patient dosing is now underway in a phase 1 clinical study of the Company's investigational systemic sclerosis treatment, bermekimab.…
According to a story from Scleroderma News, the results of a recent mouse study could have significant implications for the future of scleroderma treatment. In the study, scientists used a…
According to a story from American Nurse Today, the US Food and Drug Administration (FDA) approved a treatment on September 6th, 2019 for interstitial lung disease associated with scleroderma, a…
According to a story from Financial Buzz, the drug developer Emerald Health Pharmaceuticals, Inc., recently announced that the company had completed a phase 1 clinical trial for its experimental drug…
According to a story from Comic Sands, mother Alison Beesley is now committed to taking care of her daughters Eliza and Eleanor full time. Eliza is nine years old and…
An article from Comic Sands recently covered the story of a UK mother, Alison Beesley, who is now committed to taking care of her daughters Eliza and Eleanor full time. Eliza is nine years old and…
Dinesh Khanna, a professor at the University of Michigan has worked heavily with the rare disease scleroderma. Michigan has its own Scleroderma Program, of which Khanna is the director. Through…
According to a story from Scleroderma News, a team of researchers associated with Michigan Medicine has been awarded a grant to the tune of $10.2 million from the National Institutes…
According to a publication from Scleroderma News, a study recently published in Clinical & Transitional Immunology suggests that certain cytokines (small proteins that play an important role in communication between…
I remember when I was younger and I felt so carefree. My life was easy-breezy, and full of great times, cool friends, laughter, and a go-with-the-flow attitude. I really didn’t…
When you or your child is first diagnosed with a rare disease it can be so difficult to know where to turn. You want to learn everything about the condition,…
Scleroderma Scleroderma refers to a whole group of diseases, all rare, that cause the skin and connective tissues in the body to harden. This is a result of excess collagen…
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