According to a story from Healio, the US Food and Drug Administration's (FDA) Arthritis Advisory Committee recently voted 10-7 in favor of recommending the drug nintedanib as a treatment for…
The struggle of living with a rare disease and dealing with insurance issues was in the spotlight on national television. During Tuesday night's 2020 Democratic debate for the president, Senator…
According to a story from Angioedema News, a recent clinical trial has demonstrated that the drug conestat alfa (marketed as Ruconest) was able to successfully treat 96 percent of acute…
According to a story from the Broad Institute, there are still many mysteries to uncover related to inflammatory bowel diseases, which includes the rare form ulcerative colitis. The understanding of…
A Colorado mom is on a mission to raise money for her daughter's type 1 spinal muscular atrophy (SMA) treatment -- that would save her child’s life. Let's hear little Maisie…
According to a story from Angioedema News, a team of researchers has used data from a recent study to provide up to date recommendations for diagnosing hereditary angioedema in the…
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…
Talking about mental illness is never easy but I wanted to share my experience of it with you. In my case, it came as part of my struggle with Benign…
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Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…
According to a story from globenewswire.com, the biopharmaceutical company Mustang Bio, Inc. has recently announced that the US Food and Drug Administration (FDA) has recently awarded the company Orphan Drug…
According to a story from drugs.com, the pharmaceutical company Pfizer, Inc. recently issued an announcement detailing the approval of its drug Ruxience (rituximab-pvvr), a biosimilar to the widely used monoclonal…
According to a press release from the Kentucky-based Apellis Pharmaceuticals, the Company has initiated a program to investigate the use of APL-9, an investigational drug, in gene therapies that involve…
According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare disease…
According to a story from rediff.com, Dr. Rahul Desikan was a committed amyotrophic lateral sclerosis researcher. Then, in a cruel twist of irony, he was diagnosed with the disease himself.…
According to a story from Raconteur, as many as 3.5 million people living in the UK are expected to affected by some type of rare disease at some point during…
According to a press release from Aridis Pharmaceuticals, the Company has received Orphan Drug designation from the European Medicines Agency for its experimental cystic fibrosis-related lung infection treatment AR-501. The…
According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…
According to a publication from Charcot-Marie-Tooth News, the first patient has been enrolled in the first ever natural history study of type 4J Charcot-Marie-Tooth disease. Study collaborators — biotechnology company…
According to a publication from The Rheumatologist, the recent failure of a seemingly-promising lupus treatment in a phase 2 study has led to a discipline-wide discussion about new research and…
According to a press release from the San Francisco-based FibroGen, Inc., the first patient has been dosed in the Company's phase 3 clinical study of the experimental drug pamrevlumab for…
SMARD is such a rare disease that only 100 children in the entire world have been diagnosed with it. SMARD stands for spinal muscular atrophy with respiratory distress. SMARD…
According to a publication from Fierce Biotech, researchers from Boston Children's Hospital have created a new gene therapy technique they believe could provide benefits to patients with inherited forms of…
Patient Worthy had the privilege of attending another United Leukodystrophy Conference. The ULF’s 2019 event took place at the end of June in Illinois and had a record setting number…
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Researchers at McGill University celebrated a “first” by creating a mouse model in a dish to demonstrate the effect that mutations in the SLC9A6 gene have on brain cells.…
According to a story from EurekAlert!, in recent years immunotherapy, which are methods of treatment that enhance or harness the body's immune system to fight disease, have become increasingly prominent…
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