According to a story from globenewswire.com, ProQR Therapeutics N.V. recently announced that it has begun dosing patients in its Phase 1/2 clinical trial. This trial is testing the company's investigational…
According to a story from BioSpace, the drug developer Horizon Pharma plc recently announced the release of topline data from a Phase 3 clinical trial of its experimental drug teprotumumab,…
According to a recent article in the Sunderland Vibe, the MRC Developmental Pathway Funding Scheme (MRC) was created to carry out the work of Professor Roz Anderson, who recently died of…
According to a story from news-medical.net, a results of a recent study could help accelerate the development of therapies for liver diseases based on RNA interference (RNAi). This study demonstrated…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is the collective term used to describe ulcerative colitis and Crohn's disease. These conditions cause diarrhea, rectal bleeding, fatigue, weight loss, and rectal bleeding. These…
According to a story from the Breast Cancer Society of Canada, Veronica Dubois, who is currently pursuing a Master of Science in Medical Biophysics, is focusing her research on developing…
In the early 1900s, German psychiatrist Alois Alzheimer, while studying about the decline in brain function, recognized a correlation between behavioral changes and the buildup of tangles and plaques in…
Thanks to a grant from Worldwide Cancer Research, as well as support from various other organizations, researchers at UCLA recently uncovered a new, faster, easier, and more personalized way to…
Most biological drugs, including insulin, can't be taken orally, because enzymes and proteins cause the drugs to break down too quickly in the digestive system. For decades, the pharma industry…
According to a story from Xtalks, there has been an uptick in research related to rare diseases and the development of new therapies for them. Despite this, only a small…
Problems in China Around the world there are 7,000 rare diseases currently identified. Of these, researchers have uncovered effective treatment options for less than 10%. Sadly, even when diseases have…
SOJIA SOJIA stands for systemic-onset Juvenile Idiopathic Arthritis (JIV). It is a very severe form of JIA which causes joint pain, inflammation, recurrent fevers, and a skin rash. Patients often take predinsolone,…
According to a story from news-medical.net, a team of scientists associated with the CURECN project are starting to develop a gene therapy that has the potential to cure the rare…
Each year, Rare Disease Day (February 28th) incites individuals and organizations to investigate and reform their rare disease efforts. This year, the EU Commission has made drastic changes which should…
According to a story from Ottawa Citizen, the CHEO Research Institute has recently begun operations at its Centre for Genomic Innovation. Much of the the center's operations will focus on…
According to a story from EurekAlert!, a team of scientists has discovered a genetic variation that can cause amyotrophic lateral sclerosis. This variant acts on a biologic pathway that has…
According to a story from Gaucher Disease News, a recently published case report study suggests that instances of splenic artery aneurysm are being underreported in patients with Gaucher disease. The…
A Collaborative Effort An international team of researchers has just uncovered the genetic cause for an ultra-rare form of leukoencephalopathy. Leukoencephalopathy refers to a family of conditions which affect the…
According to a story from The Muscular Dystrophy Association (MDA), the organization alongside the Charot-Marie-Tooth Association (CMTA) has awarded grant funding totaling $276,430 to a research study that will develop…
According to a story from Statesman Journal, nine year old Haley Pollman isn't so different from most other girls her age. She loves dresses, Barbie dolls, and animals, and still…
Not only are rare diseases well, rare, but the same rare disease can also affect different patients in a multitude of ways. For instance, some diseases can impact many different…
According to a story from news-medical.net, a research team affiliated with Princeton University has been hard at work developing a unique tool that utilizes machine learning in order to help…
Ipsen has just announced that they will be acquiring Clementia Pharmaceuticals. This merging of companies is exciting for the rare community because it will strengthen the resources, knowledge-base, and overall…
According to a story from finanznachrichten.de, the biopharmaceutical company Theranexus has recently released preliminary results from a Phase II clinical trial that is testing the company's experimental product THN102. This…
According to a story from Business Insider, a recent study has found that a team of doctors has a much better chance of making an accurate diagnosis than a single…
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