A press release from Applied Genetic Technologies Corporation, which can be found in full at GlobeNewswire, has announced that enrolment for a phase 1/2 clinical study has been completed. The…
Women’s* biology and symptoms are often misunderstood in medicine, which leads to difficulties in diagnosis and treatment. Maya Dunsenbery, a journalist suffering from rheumatoid arthritis (RA), recently wrote a book,…
A young woman in Cambridgeshire received a trailblazing double lung transplant. Now she’s started a Kickstarter campaign to publish a book to help other people in similar situations, their friends…
A drug called Kineret has been approved by the European Commission in 28 European countries for the treatment of both adult-onset and juvenile-onset Still’s disease, reports Check Orphan. Still’s disease…
In an $8.7 billion agreement, Swiss drugmaker Novartis recently acquired AveXis. The company moved in such a way in order to acquire a developmental therapy for spinal muscular atrophy. The…
The University of Maryland has just published a press release after a researcher working at the university made several new discoveries about the bacteria that causes Lyme disease. Lyme disease…
According to a story from stv.tv, 14-year-old Kira Noble has been fighting against neuroblastoma for around over four years. She first began to realize that something was wrong when her…
According to a story from wired.com, a new genomics marketplace called Helix will soon start selling genetic tests directly to consumers. These tests are the same ones that doctors use,…
According to a story from globenewswire.com, the pharmaceutical companies Ultragenyx and Kyowa Kirin recently issued an announcement that the U.S. FDA has granted approval to their new treatment Crysvita (burosumab-twza).…
Pfizer has just begun a medical trial for a potential gene therapy treatment for Duchenne muscular dystrophy, and the first patient has been dosed. Click here to read the original…
According to a story from news.vanderbilt.edu, Charlotte Haffner has been working hard to advocating on behalf of amyloidosis patients. She recently underwent a heart transplant and stem cell transplant in…
According to a story from prweb.com, a possible cause of Meniere's disease may have been discovered by Dr. Michael Burcon, who has a dedicated a significant portion of his…
A $5 million charitable donation from the Isabella Santos Foundation has been committed to the Atrium Health’s Levine Children’s Hospital, reports FinacialContent. It will be used to establish The Isabella…
Researchers have found that forms of certain genes can influence how successful a blood and marrow transplant is, reports Roswell Park. This has important implications for understanding patient outcomes and…
According to a story from The Irish Times, Noreen O'Carroll can vividly remember when she first received the news that she had been diagnosed with idiopathic pulmonary fibrosis. It was…
As a female Bible college student, an outside marker of pious and godly womanhood consisted of having a male significant other. While this is not strictly a religious sentimentality about…
An online patient survey has found that 96% of pulmonary fibrosis patients, and 93% of cystic fibrosis patients, support being able to monitor their lung function at home between visits…
There is a growing consensus that patients need to be more included in the medical field, reports PCMA Convene. Over the last ten years increasing numbers of organisations have moved…
According to a recent study, cognitive behavioral therapy may be the solution for patients with misophonia, The Daily Beast reports. Misophonia, which literally means “hatred of sound,” is a rare…
It's a parent's worst nightmare-- a rare disease that strikes early in life, and without treatment, can cause a patient's death. Tyrosinemia type 1 (TYR1), is a form of a…
Eva-Rose Sturgess is eighteen months old. She faces a disease for which there is no known cure: leukodystrophy. Her parents felt like their world came apart at the seams when…
The country singer, songwriter Gavin DeGraw has released a video to raise awareness for pancreatic cancer as part of an on-going campaign by the Lustgarten Foundation, reports PR Newswire. In…
According to a story from Charcot Marie Tooth News, a small subdivision of the National Institutes of Health (NIH) is leaning into its role to assist with research into rare…
Michael Conway, 51, was diagnosed with Adrenoleukodystrophy (ALD) two years ago. Since then, he has been raising awareness for the rare and potentially life-shortening condition. He wants to let people…
According to a story from PR Newswire, the pharmaceutical company Therapix Biosciences recently announced the results of a Phase IIa study for its drug candidate THX-110. The therapy is indicated…
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