Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…
Alaina Johns, a Philadelphia based freelance writer, wears two hats. One hat as editor-in-chief of the Broad Street Review, and the other as a patient with several chronic illnesses.…
Patients with inoperable or metastasized neuroendocrine tumors (NETs) may now benefit from recently FDA approved peptide receptor radionuclide therapy (PRRT) according to a recent article in MedPage Today. The…
According to a story from GlobeNewswire, the biopharmaceutical company Onconova Therapeutics, Inc. has recently published results from a phase 1 clinical trial that was sponsored by the company. This trial…
Happy Friday! The summer heat is setting in, so why not stay in where it's cool and read the latest rare disease news. First, we want to bring attention to…
According to a story from Medscape, a recent cohort study published in the journal Cancer has concluded that updates are needed to the staging classification currently in use for renal cell carcinoma…
By Jodee Redmond from In The Cloud Copy When a patient identified as Ms. B in a case history complained of headaches starting at the back of her head and…
According to a story from thv11.com, Susan Head of Little Rock, Arkansas is a marine veteran who now faces a new battle: Castleman disease. In the midst of the ongoing…
After forty years of research to counter or cure acute myeloid leukemia and other blood cancers, a few years ago the medical community exploded with eight new cancer drugs.…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
CMT1A Charcot-Marie-Tooth disease (CMT) has a few different subtypes. 60% of all patients diagnosed with type 1 of the rare disease have CMT1A. This subtype means patients have an duplicate copy…
According to a recent press release, EspeRare received Breakthrough Therapy Designation for its investigational therapy ER-004, designed for the prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED). Ultimately, this…
According to ASH Clinical News, a phase 1 trial of lenzilumab illustrated clinical benefits for approximately one third of participants with chronic myelomonocytic leukemia. It is a novel antibody, and…
When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…
In 2016, 6-year-old Alec Ahmad loved club soccer, playing games with his two brothers, and running around with his friends at recess. He was full of energy. But every once…
A cannabinoid medication that uses a non-psychoactive ingredient of cannabis to treat rare forms of epilepsy has now been recategorized as a schedule 5 drug in the UK, the least…
At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…
Medical professionals have found that the melatonin levels of Parkinson's patients impact their symptoms, specifically the gastrointestinal, sleep disturbance, and cardiac symptoms. Parkinson's News Today reported on a study that…
Scientists and researchers have long known how important glial cells are to the nervous system. Not only do these cells make up a majority of cells within the central nervous…
A new study published by ACS Nano and conducted by scientists at the University of Illinois at Urbana-Champaign uncovered a new way to modify stem cells. This study was conducted…
Researchers at Seelos Therapeutics are planning an animal study to evaluate SLS-007, a treatment for Parkinson's disease. According to an article from Parkinson's News Today, this study is possible after…
Generating neurons is only one of many goals that have so far escaped scientists worldwide. According to an article in Yahoo, the lead author of a new study in the…
Happy Thursday! This edition of the Patient Worthy Editor's Choice is coming to you a little early this week. If you're reading from the United States, we hope you enjoy…
According to a recent article in Parkinson’s News Today, researchers at the UCSF Weill Institute discovered that disturbances in circadian rhythm increased a person’s risk of Parkinson's disease threefold. Circadian…
Scientists from the Universities of Edinburgh and Cambridge in collaboration with a research center in Germany have analyzed a DNA-damaging chemical that has given scientists a better understanding of the…
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