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A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…
Cardiff University in Wales has just announced the launch of their new Medicines Discovery Institute. It’s goal? To help accelerate the development of novel therapies for those living with conditions…
Fabry Disease Fabry Disease is a rare lysosomal storage condition. Mutations in the GLA gene cause a type of fat called globotriaosylceramide to build up in the body's cells. Symptoms…
"The simple idea of a single gene leading to a single disease is more likely to be an exception than a rule." Finding the Disease-Causing Genes Things would be, not…
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A Rare Intervention For His Children’s Rare Disease An inspiring article has recently been published in the journal Nature that credits Nick Sireau, a resident of the UK, with…
Zachary and Imogen Jones are brother and sister with cystic fibrosis (CF). While Imogen is older, she received her CF diagnosis after her brother. It was actually through educational programs…
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According to a recent article from EurekAlert!, researchers used data from the Swedish Twin Registry and found that most of the 7 diseases they were studying were inherited. The second finding…
The FDA previously announced their plan to hire 50 new employees specifically to manage the influx of gene therapy applications. Currently, they have approximately 800 applications awaiting review and they…
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Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…
According to a story from BioPharma Dive, an ongoing discussion in medical circles, both rare and otherwise, is improving the design of clinical trials. Clinical trials are an important tool…
According to a publication from Blood Journal, an experimental beta thalassemia drug called luspatercept yielded positive results at the end of a phase 2 clinical study. Many patients enjoyed an…
According to a story from Korean Biomedical Review, a team of researchers from Seoul National University, headed by professor Paek Sun-ha, have developed an approach utilizing 3D cell printing technology…
A recent generous donation from the Michael J. Fox Foundation for Parkinson's Research (MJFF) will help develop a novel piece of technology which could accelerate the research of new treatment…
As the field of medicine has continually advanced, more and more children (now approximately 80%) are surviving cancer diagnoses. It's amazing. It also means that researchers are able to focus…
Acute Myelogenous Leukemia Acute myelogenous leukemia (AML) is a rare blood cancer whose prevalence increases with age. There are still extremely limited treatment options for AML and the five-year survival…
A recent study published in Cell has exhibited the scientific community's negligence for conducting research that includes an adequate representation of non-Europeans. Specifically, it showed that 78% of patients in genomic…
Bruce Alan Fries, President of the Patient Centered Care Advocacy Group, along with Holly Ahern and Chris Fisk from the Lyme Action Network, have issued an official "Information Quality Request…
According to a story from BioSpace, the biotechnology company Spruce Biosciences recently announced that they have achieved positive proof of concept data in the company's clinical trial. This is a…
A preliminary study has found that dementia may only affect select areas of the brain in the early stages. Through this research, they've also uncovered what they believe is a…
The National Institutes of Health (NIH) is the primary medical research institution in the United States. The NIH is one of several public health institutions, like the Food and Drug…
According to a story from BioSpace, a recent study revealed that hemophilia A patients were less likely to experience bleeds when they used new, extended half-life therapies on a preventive…
According to a press release written by French biotech company Deinove and published by PM360, the pharmaceutical manufacturer stands ready to begin phase 2 testing into their experimental Clostridium difficile…
The longer a patient sees a doctor, the more their initial trust, confidence, and comfort with them grows. This is amplified for patients living with rare conditions as doctors visits…
According to a story from The McGill Tribune, a little girl named Mathilde Poliquin recently died at the Montreal Children's Hospital. She was only two and half months old and…
According to a story from PR Newswire, the biopharmaceutical company Therachon AG recently issued a presentation of results from the company's Phase I clinical trial. This clinical trial tested the…
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