Besides the obvious need for ties within the medical, pharmaceutical, and social industries, I have always wondered internally why it is so necessary that parents, adults, children, and loved ones…
Gastroschisis Study Cincinnati Children’s Medical Center, Texas Children’s Hospital, and Avery’s Angels are working together to try to learn more about the long-term gastrointestinal issues that people born with gastroschisis…
According to a story by Mirror, the Rare Disease Centre at Birmingham Children’s Hospital is set to begin operations after a massive donation by readers of The Star Appeal. Readers…
Dr. David Scadden is a hematologist, oncologist, and Harvard medical school professor. He is also the author of a memoir for a book recalling his early medical schooling and career.…
According to a story from Benzinga, the pharmaceutical company BioCryst Pharmaceuticals, Inc., recently announced that its investigational product BCX7353 has been granted Fast Track Designation from the FDA for the…
According to a recent article for myscience.org, there is new potential for genetic therapy to cure the often deadly neurodegenerative disorder known as Gaucher disease. The new studies were first…
These groundbreaking research results have been published on the website of the University in Erlangen and in the New England Journal of Medicine, and I would like to share them…
We're happy to introduce a new partner to Patient Worthy's mission of advocating for rare disease patients! The Aplastic Anemia and MDS International Foundation (AAMDS) supports, connects and educates patients, caregivers…
According to a story from MPR News, Rae Baylark, who is the founder and current president of the Sickle Cell Foundation of Minnesota, felt like she had nowhere to seek…
According to a report by Science Daily, researchers recently discovered a disease in monkeys which imitates human Batten disease. Establishing the closest analog to the condition in humans, the version…
According to a Science Daily report, new research by a group in Liverpool recently uncovered a drug for the treatment of alkaptonuria. Researchers published the collaborative study in the journal…
According to a story from the pharmaceutical company Takeda, the company recently released the results from its clinical trial testing an experimental subcutaneous formula of the drug vedolizumab. The drug…
A Phase 1/2 clinical trial of the experimental drug NV1205 in patients with childhood cerebral adrenoleukodystrophy (CCALD) has been initiated, announced NeuroVia Inc. The source article can be found here,…
According to a story from Beyond the Dish, a new experimental treatment for spinocerebellar ataxia was given the go-ahead from the U.S. Food and Drug Administration for testing. The investigational…
According to a story from BioPortfolio, the company Mologic Ltd recently announced that its clinical trial to test HeadsUp, its urine-based diagnostic tool, is starting. The clinical trial will specifically…
According to a source article that can be found here on Ultragenyx’s website, the first patient has been dosed in a trial of an investigational gene therapy called DTX401 that…
A study is planned to investigate the effects of the experimental drug PTC596 in children with recently diagnosed diffuse intrinsic pontine glioma and high-grade glioma. The study is made up…
The first patient has been treated in the Empowers study, a clinical trial investigating an experimental gene editing therapy for the treatment of mucopolysaccharidosis type I. The source article, which…
According to a story from Buffalo News, Brett Perla was graduating high school when he first starting to experience symptoms of a rare condition of the cornea called keratoconus. This…
According to PRNewswire, Beverly Hills and Frankfurt based INFUSIO donated $5,000 each of their patient’s Lyme disease treatment in May. Interest in the program was high. As a result, INFUSIO…
According to University of Wisconsin News, UW-Madison professor David Beebe and his colleague Anna Huttenlocher, fellow UW professor, were awarded a $3.7 million grant from the National Institutes of Health (NIH)…
The Foltz family relates to a number of usual, happy family evenings. Some of them, however, include unnerving moments in which their son, Trevor, experienced multiple seizures. At first they…
According to a story from PR Newswire, the U.S. Food and Drug Administration had approved the New Drug Application for an innovative new delivery system for Remodulin, a therapy that…
July 13th was Gastrointestinal Stromal Tumors Awareness Day! And that "13" has extra relevance. 5,000 Americans are diagnosed with Gastrointestinal Stromal Tumors (GIST) each year. That’s 13 people diagnosed each…
According to Business Wire, a pharmaceutical company, Aurinia Pharmaceuticals Inc., recently announced that they have enrolled the first lupus nephritis patients in the AURORA 2 extension study. Not only this, but Aurinia…
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