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…
Sound Pharmaceuticals announced the first-ever Phase III clinical trial protocol for Meniere’s disease to be approved by the FDA. Meniere’s disease (MD) is a neurotologic disorder that involves tinnitus (ringing…
A study in the Journal of Movement Disorders on patients with Parkinson's disease has found a relationship between sedentary living and attention difficulty. Exercise is the age old medicine, with…
A recent study has demonstrated that through standardizing the whole genome sequencing process and making it routine for patients, we can improve rare disease diagnosis across the board. Earlier diagnosis…
Emily Ventura was already well aware of the risk of germs before coronavirus. She works as a nurse in the ICU and takes care of her 8-year-old, who is immunosuppressed.…
According to a recent article in The Guardian, funding received from the NIHR and Medical Research Council will facilitate the launch of the largest genetic study of chronic fatigue syndrome…
uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington's disease. Two patients are included so far, with one being given…
Researchers believe that they may have found the cause of myasthenia gravis (MG), according to an article in Myasthenia Gravis News. A study found that issues with mitophagy in immune…
Global Blood Therapeutics (GBT) will submit their Marketing Authorization Application (MAA) for Oxbryta to the EMA by 2021, says a recent BioSpace press release. Ultimately, the MAA will seek regulatory…
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