According to a story from EurekAlert!, the results from a recent Phase 2 clinical trial suggest that a two drug combination could be a useful approach for people with rare,…
Lambert-Eaton Myasthenic Syndrome (LEMS) is an autoimmune disorder. It's a rare condition characterized by muscle weakness in the limbs. When the muscle weakness includes the respiratory muscles, the disease can…
According to a story from Medical Xpress, a team of scientists affiliated with the University of Granada have developed a new potential treatment for a form of mitochondrial disease that…
According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in…
Stand Up To Cancer (SU2C) has donated a million dollars for a research project dedicated towards a new way of deploying the immune system against pancreatic cancer. This is welcome…
According to a story from PR Newswire, the biopharmaceutical company Constant Pharmaceuticals recently announced that it plans to develop its experimental compound TXA127 as a therapy for epidermolysis bullosa (EB).…
On November 26, 2018, the FDA approved a new autoinjector for individuals with severe rheumatoid arthritis (RA), giant cell arteritis, and polyarticular or systemic juvenile idiopathic arthritis (aged 2 and older). It's…
On November 26, 2018, a new drug was given accelerated approval by the FDA. Not only that, but it was granted orphan drug designation and breakthrough therapy designation. The drug…
For many families, when their child is born with developmental delays, there may be weeks, months, or even years that go by without a diagnosis. During this time, parents…
Lynch Syndrome Lynch syndrome is a genetic condition which makes an individual more susceptible to certain types of cancers. Specifically, it has been thought to increase risk for developing colorectal…
Niemann-Pick Disease Type C is a lysosomal storage disorder caused by malfunction of the NPC1 protein. This protein's dysfunction results in a buildup of lipids in the lysosomes. As a…
According to a story from BioPortfolio, Celgene Corporation and bluebird bio, Inc., have recently announced that enrollment for a pivotal Phase 2 study testing an experimental therapy for relapsed/refractory multiple…
According to a story from MarketWatch, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., recently presented interim data from a Phase 2 trial of setmelanotide as a treatment for obesity associated with…
According to a story from Parkinson's News Today, more and more research is indicating the exercise can have many significant benefits for Parkinson's disease patients. While there are no therapies…
According to a story from globenewswire.com, the biopharmaceutical company PhaseBio Pharmaceuticals has recently announced that the first patient has been dosed in a Phase 2b clinical trial of the company's…
The following story includes discussion of suicide. According to a story from EurekAlert!, a recent study has revealed that survivors of head and neck cancer are four times more likely…
In 2013 researchers discovered something interesting about the protein BH4. They found that by inhibiting the protein they could reduce patient's pain. A drug was created called Quartet, and it…
According to a story from Business Wire, a recent survey has revealed new findings about the frequency of neurogenic orthostatic hypotension (nOH) among Parkinson's disease patients. Around one in five…
According to a story from MedicalXpress, it was around 30 years ago when two sons of Marsha and Allen Barnett, named Chuckie and Michael, both passed away from Leigh syndrome.…
According to a story from tgen.org, a team of scientists from the Translational Genomics Research Institute (TGEN) have recently initiated a study that will test the effectiveness of liquid biopsy…
According to a story from Health Canal, a team of researchers led by Dr. Mohammad Siddiqui of Virginia Commonwealth University (VCU) recently released a study which further highlights the relationship…
4D110 has just received Orphan Drug Designation for the treatment of Choroideremia (CHM). The phase 1 trial of the drug is expected to start in 2019. What is Chorioderemia? Chorioderemia is…
According to a story from uhb.nhs.uk, a recent study has drawn a link between chronic kidney disease and Alström syndrome. The research team is associated with the University Hospitals Birmingham…
A frequent impediment to rare disease research is cost. It's unfortunate, but it's the reality of the world we live in. That means every fundraising campaign and every dollar raised…
According to a story from Scimex, the Children's Medical Research Institute in Sydney, Australia, has recently agreed to a new partnership with LogicBio Therapeutics, a biotechnology company from Boston, MA.…
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