UPDATE: Patisiran has been approved! Check out the story here. According to a story from Xconomy, the U.S. Food and Drug Administration could be close to approving a new drug…
Ehlers-Danlos syndromes (EDS) are a group of rare conditions that affect the connective tissue and can be inherited. Connective tissue lies between tissues and organs throughout the body and helps…
At the 2018 Aplastic Anemia and MDS International Foundation’s (AAMDSIF) International Bone Marrow Failure Disease Scientific Symposium of March 2018, Dr. David Margolis from the Medical College of Wisconsin, and…
In an article by the Fibrous Dysplasia Foundation, the brief and wondrous life of Mauricio Saravia is remembered and celebrated on the advent of his mother’s memoir publication. Mauricio,…
According to a source article on Business Wire that can be found here, the Committee for Orphan Medicinal Products (part of the European Medicines Agency) has recommended that the experimental…
According to a story from PR Newswire, the specialty drug development company Mallinckrodt recently announced that enrollment for trials of its experimental medication MNK-1411 has started. The drug will be…
According to a report by EuroInvestor, biopharmaceutical company Novelion Therapeutics, Inc. was recently granted permission to market their drug MYALEPTA. Novelion intends MYALEPTA to function as a method of treating…
According to a story from Market Screener, the biopharmaceutical company MediciNova, Inc., recently announced its intention to begin a Phase 2/3 clinical trial for its product MN-166. MN-166, also known…
Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease. With fetal gene…
According to a story from businesswire.com, Otsuka Pharmaceutical Europe Ltd recently announced that its product JINARC, also known as tolvaptan, now has an expanded therapeutic indication after a recent approval…
Patient Worthy exists because there's just not enough visibility for the nearly 30 million Americans who have been diagnosed with disorders that are rare or have limited treatment options. So…
According to a story from BioSpace, an announcement from the biotechnology company Vertex Pharmaceuticals revealed that the U.S. Food and Drug Administration has approved an expanded indication for ORKAMBI, the…
Diseases affecting the brain are often pose numerous difficulties in research. One such challenge is that animal models of the diseases never quite replicate the human reality. In mice, for…
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…
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