According to a story from Specialty Pharmacy Times, data from a Phase II clinical trial has recently been released which suggests that the drug tagraxofsup is a useful treatment in…
Diffuse Intrinsic Pontine Glioma Diffuse intrinsic pontine glioma (DIPG) is a rare form of childhood brain cancer that has a 100% mortality rate. Children diagnosed with DIPG only typically live…
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A recent article in the neuroscience section of the Medical Express reports the results of a study conducted by researchers in the Charcot-Marie-Tooth/Hereditary Neuropathy Center at internationally acclaimed Cedars-Sinai.…
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It has been less than two years since Cytokinetics’ previous ALS muscle drug, tirasemtiv, failed to meet its endpoint in a Phase III trial to treat ALS. (That was after…
Researchers at the University of Colorado Anschutz Medical Campus have uncovered a never-before discovered immunodeficiency disease. How It Came About It started with a newborn boy who had a myriad…
According to a story from Biopharma Dive, an investigational therapy for Huntington's disease that is in development could be the first step in the implementation of the first-ever disease modifying…
Mantle cell lymphoma (MCL) and acute myeloid leukemia (AML) are two rare forms of blood cancer with a high unmet need. The majority of cases in these patient populations are…
Unknown Side Effects It's almost as if it's just a matter of time before a safety warning is issued for each new drug approved by the FDA stating an unexpected…
According to a story from globenewswire.com, the drug developer Zynerba Pharmaceuticals recently announced that the company has been awarded Fast Track Designation from the US Food and Drug Administration (FDA).…
According to a story from Medical Xpress, a recent study has suggested that the drug eculizumab could substantially reduce the risk of disease relapse in patients with the rare disease…
According to a story from BioSpace, the drug development company Dynacure recently released an announcement in which the company stated that its application for a Phase 1/2 clinical trial had…
According to a story from sciencemag.org, an experimental gene therapy for the rare genetic disorder X-linked myotubular myopathy is starting to produce significant improvements for patients. A devastating disease that…
Transthyretin-Mediated Amyloidosis Cardiomyopathy The FDA has just approved the very first treatment for hereditary or wild-type transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM). ATTR-CM is a rare disease that occurs when amyloids, an…
According to a story from Science Daily, a team of scientists affiliated with UT Southwestern's Children's Medical Center Research Institute are hard at work pioneering an innovative new approach that…
A recent report that was published in the Swiss Medical Weekly outlines data gathered by a Swiss panel of experts on plasma cell myeloma. The report offers an update and…
The 2019 Congress of Clinical Rheumatology held May 2nd through the 5th in Destin, Florida featured a talk by Yusuf Yazici, the Director of the Behçet Syndrome Center at the…
According to a story from fox43.com, an experimental therapy for the genetic form of amyotrophic lateral sclerosis (ALS) has been termed "game changing." A study that will soon be presented…
According to a story from EurekAlert!, the biopharmaceutical company Celgene Corporation and the gene therapy company bluebird bio, inc. recently announced that the interim results for a Phase 1 clinical…
Chronic Fatigue Syndrome Chronic fatigue syndrome (CFS) is a rare disease which causes, most notably, persistent fatigue that doesn't improve with rest. It is also sometimes referred to as myalgic…
According to a story from globenewswire.com, the pharmaceutical company Acer Therapeutics recently announced the release of data from a long-term observational study of patients with vascular Ehlers-Danlos syndrome (vEDS). All…
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The News Hub at Washington State University recently carried an article describing initial success by researchers towards developing a drug for a rare form of muscular dystrophy. Several thousand people…
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Researcher: “MND is not an incurable disease. It is an underfunded disease” The Trinity Times recently interviewed Prof. Orla Hardiman on the subject of motor neuron disease (MND), also…
According to a story from finanzen.at, a recent early stage clinical study showed positive results from an innovative stem cell therapy treatment. In the study, this therapy was able to…
According to a story from Ankylosing Spondylitis News, a recent study has reaffirmed the understanding that patients with ankylosing spondylitis, a rare form of inflammatory disease that affects the joints…
According to a story from Lambert-Eaton News, a recent large scale study using data from the Veterans Affairs has determined that the prevalence of Lambert-Eaton myasthenic syndrome (LEMS) is similar…
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