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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A recent posting by the Leukemia and Lymphoma Society references the Beat AML Master Trial. The trial evaluates targeted therapies to treat acute myeloid leukemia (AML) and was launched…
Uveitis Uveitis is a rare condition in which the uvea of the eye becomes inflamed. The uvea is responsible for providing the majority of the blood to the retina and…
According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular…
After 20 agonizing years, a mother and father have finally received answers regarding their daughter's health. It shouldn't take this long. The Diagnosis Their daughter was diagnosed with two very…
According to a story from Healio, a recent study has found that 58 percent of patients with the inflammatory disease ankylosing spondylitis will experience relapse within one year after stopping…
According to a story from finanzen.ch, the pharmaceutical company Eisai, Inc. announced that that company presented a total of four abstracts which were related to a Phase 3 clinical trial…
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