According to a story from BioSpace, the biopharmaceutical company Immunic, Inc. has announced that the first patient has signed up for its proof-of-concept clinical study. This study is intended to…
According to a story from Medpage Today, a recent phase II clinical trial of an experimental dual-action CAR-T cell therapy has yielded an impressive 95 percent response rate. The drug…
According to a story from Express Digest, 22 year old Steph Todd first started experiencing symptoms of Lyme disease in 2015, when she presented with flu-like symptoms and a circular…
In a story reported in the Boston Globe, Stephen Marcus, as a person with Crohn’s disease, knows the laws surrounding bathroom policies from public franchises. He worked as a lawyer…
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Popular Science published an article recently about the first time an attempt was made to treat sickle cell anemia using CRISPR technology. The patient, Victoria Gray, age 41, from…
At the heart of rare disease treatments is research. Currently, 95% of all rare diseases still have no approved therapies. Although there has been an increased focus on rare diseases…
The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…
According to a story from Medpage Today, a retrospective study has identified signs that can predict whether a patient has a significant risk of developing severe, aggressive multiple sclerosis. These…
Hey Patient Worthy community - let's help a young girl with aplastic anemia find a bone marrow donor! Nine-year-old Emma Hall needs our help - so let's see what we…
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Trust is vital when it comes to medical care. People put their most important questions and qualms into the hands of doctors and medical staff and expect that they can…
According to a story from MD Edge, a phase 2 clinical trial testing the drug concizumab as a treatment for hemophilia A and hemophilia B demonstrated substantial capability in reducing…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is actually a term used to classify a few different conditions. Two of these are Crohn's disease and ulcerative colitis. These are rare…
Juvenile idiopathic arthritis (JIA) is a rare condition affecting children. Like all patients, these children are told what treatment they need and then they are expected to comply. However, researchers in…
According to a story from Trinity News, a team of researchers with the Academic Unit of Neurology at Trinity College Dublin have recently conducted a study that maps the ways…
According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…
Usually when people think of a seizure disorder, they think of something called tonic-clonic seizures or episodes where a person loses consciousness, has muscle stiffness, spasms, and jerky movements.…
According to a story from news.cision.com, the Swedish biotechnology company Cyxone recently announced positive results from the company's phase 1 clinical trial. This trial was testing the tolerability and safety…
According to a story from globenewswire.com, Akcea Therapeutics and Ionis Pharmaceuticals recently announced the release of top line results from a clinical study. This trial tested the drug WAYLIVRA as…
According to a story from globenewswire.com, the drug company Zynerba Pharmaceuticals, Inc. has announced that the data from its phase 2 open label trial has been officially published. This clinical…
According to a publication from Duke University, Dr. Karissa Gable, a neurologist at the University, has received a one-year research grant from the GBS|CIDP Foundation International to study the activity…
According to a story from Connecticut Children's, the hospital is one of the very first in the country to administer Zolgensma, a recently approved gene therapy for the rare genetic…
Lucy is now ten years old and stable, living her life as close to a normal ten-year old as possible. However, she was born with a super rare disease…
According to a story from fda.gov, the US Food and Drug Administration (FDA) has approved the drug pexidartinib (marketed as Turalio) as a treatment for tenosynovial giant cell tumor (TGCT)…
According to a publication from Scleroderma News, a recent study suggests that blood concentrations of certain immune cells could correlate with the severity of cases of systemic sclerosis. About Systemic…
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It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
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