According to a story from EurekAlert!, a team of researchers affiliated with UT Southwestern were successfully able to curtail inflammation-associated colorectal cancer in mice by precisely editing the animals' gut…
According to a story from EurekAlert!, the results of a recent clinical trial should get the attention of patients in the US with the rare disorder familial chylomicronemia syndrome. The…
According to a story from Newswise, Quest Diagnostics, a laboratory that specializes in diagnostic information, has announced plans to participate in a unique, large scale trial called NCI-MATCH, which stands…
Critical Path Institute The Critical Path Institute, or C-Path is a nonprofit organization which was established in 2005. It has a US branch, headquartered in Tucson, Arizona as well as…
Epidermolysis Bullosa Epidermolysis bullosa (EB) is a rare condition affecting approximately 500,000 individuals globally. Patients in the United States and Europe account for 50,000 of these individuals. The hereditary condition is…
According to an article from journals.lww.com, the treatment of mantle cell lymphoma, a rare type of blood cancer, has historically been a significant medical challenge. However, recent developments in the…
According to a story from Medpage Today, a recent study testing the medication abatacept (marketed as Orencia) as a treatment for diffuse cutaneous systemic sclerosis concluded with rather mixed results.…
According to a story from globenewswire.com, gene therapy, a current hot topic in the medical field, is continuing to make headlines. The drug company Amicus Therapeutics recently announced that a…
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It's common knowledge among researchers who study diseases called toxic proteinopathies that this group of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
As covered in Stat and True Viral News, ALS made a name for itself in 2014 when their donations campaign, the ice bucket challenge, went viral, earning $115 million dollars…
According to a story from globenewswire.com, the drug developer ProQR Therapeutics N.V. has recently announced that the US Food and Drug Administration (FDA) has officially cleared the company's Investigational New…
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Patient Worthy wishes to share the following funding opportunities with faculty/ biotech having interest in these areas of research. The two grant programs were announced by the Orphan Disease Center…
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…
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