According to a story from The Motley Fool, The gene therapy development company bluebird bio has been working on a treatment for beta thalassemia, and it has the potential to…
AKU Society is hosting it's 2nd International Patient Workshop in Liverpool's Bluecoat. The event is intended to educate about new scientific research, clinical trials, diet guidance and more. RVSP to…
Happy Friday Everybody! This week we're spotlighting three patient stories: an ALD patient full of optimism and love, a beauty pageant contestant with a noble goal, and a woman with…
According to a story from biotechconnectionbay.org, medical researchers are beginning to more closely investigate that possibilities that drug repurposing, or using old drugs for treating different illnesses, could offer. While…
According to a story from statnews.com, a drug derived from substances originating in the cannabis plant may be nearing approval by the U.S. Food and Drug Administration. The drug is…
The pharmaceutical company Lonza has just opened a new state-of-the-art manufacturing facility to produce cell and gene therapies. You can find the full press release here, on Lonza’s website.…
According to a story from Summit Medical Group, a recent study indicates that survivors of testicular cancer may have a greater risk for heart disease. Compared to many types of…
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 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 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…
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…
According to a story from medpagetoday.com, researchers are beginning to realize that symptoms of pediatric spondyloarthritis present themselves in a distinct way when compared to the symptoms that are experienced…
Hope everybody is enjoying the warm weather! This week we have patient stories about a woman with MS who started a support group and a rare mom who took issues…
According to a story from QQ Malaysia Online, patient advocates are looking for ways to raise awareness of rare diseases in Malaysia and get help to forgotten patients that desperately…
According to a story from scimex.org, a recent data review is calling into question the previously held consensus that common pain medicines such as NSAIDs and paracetamol could make inflammatory…
No matter which way you spin it, Ehlers-Danlos syndrome (EDS) is generally not a great time. EDS is a rare genetic condition that affects connective tissue throughout the body. It…
According to a story from ankylosingspondylitisnews.com, a significant proportion of patients stopped taking a biosimilar for infliximab within six months of transitioning to the new medication. This suggests that the…
According to a story from The Irish Times, a new campaign is encouraging people with the rare lung condition idiopathic pulmonary fibrosis to keep exercising. The campaign is centered around…
A recent story from Science Daily is highlighting just how difficult it can be for patients with rare diseases to get an accurate diagnosis. For patients with mitochondrial disease, the…
According to a story from Neurology Advisor, a retired physician experienced the challenges of being a rare disease patient first hand after being diagnosed with primary periodic paralysis thanks to…
According to a recent story from pm360online.com, the pharmaceutical company Shire announced that the marketing application for its drug product lanadelumab has gained approval from the European Medicines Agency (EMA).…
According to a story from Newswise, researchers at the University of Texas Southwestern Medical Center are reporting the a new drug developed for the treatment of rare, pediatric cancers, has…
According to a story from the Independent, father Nick Sireau is worried that Brexit, the United Kingdom's plan to withdraw from the European Union, could affect the ability of his…
According to a story from 4-traders.com, the pharmaceutical company SteroTherapeutics recently announced that its drug candidate ST-002 was awarded Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).…
According to a story from rte.ie, a team of scientists from the Trinity College Dublin have managed to discover a new technique that allowed them to shut down inflammatory response…
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