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…
Famous poetic thought suggests that "the eyes are the window to the soul." Doctors studying Wilson disease, however, may see the eyes as the window to a diagnosis. According to…
According to UPI, a recent study has found that standard Parkinson's disease treatments may be responsible for a plethora of other things as well. Most notably, those who take…
According to a report by The Herald Scotland Online, a landmark study recently reveled flight crew to be exposed to greater than average risk of thyroid cancer. Surprisingly, the results…
What could make a family in Lebanon travel all the way to UCLA? The answer is a unique stem cell gene therapy designed to combat SCID. The El Kerdi family…
According to a report by Evening Times, two new drugs targeting Huntington’s disease are on the way. Developed by Wave Life Sciences, the new drugs will soon undergo clinical trials…
According to a story from philly.com, four year old Ethan Lieber of the Roxborough neighborhood in Philadelphia is one of just 500 people in the US with Hunter syndrome. This…
Who couldn’t use a little extra money now and again? Better still, what if earning that money was as easy as a mouth swab? According to a report on Journal…
According to a story from the FH Foundation, the non-profit organization has released a vital consensus statement regarding the diagnosis of familial hypercholesterolemia, which is the most prevalent genetic cause…
PTEN Hamartoma Tumor Syndrome (PHTS) What is PTEN Hamartoma Tumor Syndrome (PHTS)? PTEN hamartoma tumor syndrome (PHTS) is an umbrella term referring to a spectrum of conditions that are all…
To read parts 1 and 2 of Maxine's dysautonomia journey, click here and here. It was a complete scope of my upper gastro track and everything went well with the…
According to a story from WebMD, a recent study concludes that the rates of diagnosis for thyroid cancer have been steadily increasing recently, and that it is over treated in…
According to a story from cbc.ca, Cole Pringle is a 31-year-old man from Regina, Saskatchewan with spinal muscular atrophy, a debilitating illness that severely affects his mobility. He was diagnosed…
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