Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.
9% Copyscape An investigational drug called CLR 131 has received Rare Pediatric Disease Designation from the US FDA for the treatment of Ewing’s sarcoma. The source press release can be…
A research team led by scientists from the Beth Israel Deaconess Medical Center (BIDMC) have found that arsenic, in combination with the drug all-trans retinoic acid (ATRA), may have the potential…
The award-winning actor Alan Alda has shared his diagnosis of Parkinson’s disease during an interview on the CBS This Morning show. The source video of the interview can be found here on…
A recently published study in the Journal of Biosciences and Medicines has concluded that orthoses may help to improve the gait and balance of patients with Charcot-Marie-Tooth disease. Orthoses are…
A recent study into Periodic Fever, Aphtous stomatitis, Pharyngitis and Adenitis (PFAPA) syndrome investigated the impact of the condition on children and found that is was associated with a reduction…
A recently published paper called ‘Biallelic loss of human CTNNA2, encoding αN-catenin, leads to ARP2/3 complex overactivity and disordered cortical neuronal migration’ outlines new research into the genetic basis of…
Gemphire Therapeutics Inc. has been recommended to stop a clinical trial by the Data and Safety Monitoring Board at Emory University School of Medicine. The clinical trial, which ran into…
The company Epizyme has announced the results of a Phase 2 clinical study of its lead candidate drug, tazemetostat, in the treatment of relapsed or refractory malignant mesothelioma with loss…
A motor neurone disease clinical drug trial will take place in Scotland for the first time in more than twenty years. This study is the first step in a broader…
The top-line results of a pivotal Phase 3 study investigating the drug recorlevTM (levoketoconazole) in patients with endogenous Cushing’s syndrome have been released. The source article, which contains more detailed information,…
An investigational drug called ALT-P7 is being developed as a potential medicine for certain forms of cancer. The drug has received Orphan Drug Designation for the treatment of gastric cancer…
The US FDA has awarded Orphan Drug status to the investigational drug NSI-189 for use by people with Angelman syndrome. You can read the source article here, at Nasdaq. About…
The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis.…
The European Medicines Agency has awarded Orphan Drug designation to selumetinib for the treatment of neurofibromatosis type 1. You can read the source press release here, on Merck’s website. About…
Ehlers-Danlos syndromes (EDS) are a group of rare conditions that affect the connective tissue and can be inherited. Connective tissue lies between tissues and organs throughout the body and helps…
According to a source article on Business Wire that can be found here, the Committee for Orphan Medicinal Products (part of the European Medicines Agency) has recommended that the experimental…
According to a source article that can be found here on Ultragenyx’s website, the first patient has been dosed in a trial of an investigational gene therapy called DTX401 that…
A study is planned to investigate the effects of the experimental drug PTC596 in children with recently diagnosed diffuse intrinsic pontine glioma and high-grade glioma. The study is made up…
The first patient has been treated in the Empowers study, a clinical trial investigating an experimental gene editing therapy for the treatment of mucopolysaccharidosis type I. The source article, which…
A series called ‘Gonads’ by RadioLab is looking into human reproduction. Their second episode, which is the source used for this article, can be found here. It explores the story of Greg…
A study of Lynparza (olaparib) in combination with abiraterone for the treatment of metastatic castration-resistant prostate cancer has shown promising results. The full article can be read here, at Bio…
Results from two studies looking at the effects of an experimental drug (bardoxolone) on kidney function in patients with chronic kidney disease (CKD) have been released. One of the studies…
An ongoing Phase 3 clinical trial program is investigating the drug PXT3003 as an experimental treatment for Charcot-Marie-Tooth disease type 1A in adults. Pharnext SA says that they expect to…
The first patient has been enrolled onto a Phase 3 clinical trial investigating topical dermal reproxalap as a possible treatment for Sjögren-Larsson syndrome associated ichthyosis. The original article can be read…
Researchers have investigated whether self-management electronic health (eHealth) interventions, such as text messaging, apps, and online cognitive-behavioural therapy, should be used to help patients with sickle cell disease self-manage the…
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