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.
Military veterans are known to have twice the risk of developing amyotrophic lateral sclerosis (ALS) compared to the general population, but researchers are yet to understand why. The full story can…
Continue ReadingMilitary Veterans are at Twice the Risk of Developing ALS
The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to the experimental drug Lenti-D, which is being produced by the gene therapy company Bluebird bio to treat cerebral…
Continue ReadingAn Experimental Gene Therapy for Cerebral ALD Has Been Granted Breakthrough Therapy Designation by the FDA
The not-for-profit healthcare organisation Sanford Health is planning to implement routine genetic testing for patients to identify a patient’s risk for a range of rare, and more common, diseases. The…
Continue ReadingRoutine Genetic Screening for Diseases is Being Implemented by Sanford Health
In the Victorian period, the British Royal family was known to carry haemophilia, leading to the condition also being known as ‘the royal disease’. Now, no members of the British…
Continue ReadingThe British Royal Family’s History with Haemophilia
The Young family from Oregon, U.S. have used genetic screening to test their embryos for a variation of the BRCA gene that significantly increases the risk of breast cancer, reports…
Continue ReadingGenetic Screening Can Prevent Parents from Passing on Genes Linked to Some Diseases
The Melanoma Research Alliance (MRA) and The American Cancer Society (ACS) have announced the first recipients of their new research grants, reports PRNewswire. The grants have been set up as…
Continue Reading$2.6 million has Been Granted to Researchers Working on Reducing the Side-Effects of Checkpoint Inhibitor Immunotherapies For Cancer Patients
A new treatment for cystic fibrosis has received a lot of media attention in the UK after the National Health Service (NHS) chose not to fund it due to its…
Continue ReadingCystic Fibrosis Advocates are Petitioning the UK Government to Fund the Drug Orkambi
Researchers at Columbia University have found a way to apply CRISPR, a tool used in gene therapies, to new types of disease, reports News Medical. The study was carried out…
A new documentary, Unrest, explores myalgic encephalopathy (ME), also known as chronic fatigue syndrome. The film was premiered at the Sundance Film Festival 2017 where it received an award for…
Continue ReadingUnrest: The New Documentary about Myalgic Encephalopathy (Chronic Fatigue Syndrome)
A company called Beam Therapeutics is developing a new form of genetic editing, called ‘base editing’, to treat certain genetic diseases. The full story can be read here, at MedCity…
Continue ReadingA Startup is Developing More Precise Gene Therapies
The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug Sarconeos, developed by the company Biophytis, for the treatment of Duchenne muscular dystrophy. The…
Continue ReadingAn Experimental Drug for Duchenne Muscular Dystrophy Has Been Awarded Orphan Drug Status by the F.D.A.
Researchers have found a new way to monitor disease progression in people with Charcot-Marie-Tooth disease type 1 (CMT1), reports Charcot Marie Tooth News. CMT1 is an inherited genetic condition that…
Continue ReadingResearchers Are Investigating a New Way to Monitor Disease Progression for People with Charcot-Marie-Tooth Disease Type 1
A pre-clinical study of a treatment for Rett syndrome has produced encouraging results, and it is hoped that this will lead to further research. The full story can be read…
Continue ReadingA Pre-Clinical Study on Rett Syndrome Has Produced Promising Results
A fourteen-year-old girl called Kira Noble from Edinburgh, Scotland has raised £340,000 (about $460,000) for surgery to remove her neuroblastoma tumour, reports The Daily Record. Kira was first diagnosed with…
Continue ReadingA Girl From Scotland Has Raised £340,000 For Neuroblastoma Surgery
Collaboration between the FDNA, the Cure Sanfilippo Foundation, and the Jonah’s Just Begun Foundation has led to technology that successfully recognises the facial phenotype (observable characteristics) of patients with mucopolysaccharidosis…
Continue ReadingAn Algorithm That Uses Facial Features to Diagnose Sanfilippo Syndrome Type-B Has Been Developed
The pharmaceutical company Biohaven has announced that they will be using an expanded access program to make an experimental drug for amyotrophic lateral sclerosis (ALS) available to patients before F.D.A.…
Continue ReadingAn Experimental Drug for ALS Patients Will Become Available Through an Expanded Access Program
A recent study published in the Lancet has investigated the effects of starting chemotherapy on patient survival, according to the UK Government website. It was found that many factors can…
Continue ReadingA Large-Scale Study on Chemotherapy Identifies Risk Factors For Patients
Scientists working at the Broad Institute of MIT and Harvard are trying to build a collection of every drug ever developed. They are planning on using the collection to re-purpose…
Continue ReadingResearchers are Collecting All The Drugs That Have Ever Been Developed to Find New Treatments For Diseases
A clinical trial that is investigating a drug designed to treat ulcerative colitis has completed patient enrolment, reports Business Wire. Ulcerative colitis is a long-term inflammatory bowel condition. The severity…
Continue ReadingA Clinical Trial Investigating a Drug for Ulcerative Colitis Has Finished Enrolment
A team of researchers at Imperial College Lonon in the UK have carried out studies that suggest it may be possible to use gene editing to treat pancreatic cancer, reports…
Continue ReadingResearch into Gene Editing as a Treatment For Pancreatic Cancer Has Shown Positive Results
The U.S. Food and Drug Administration (FDA) has just approved a subcutaneous version of a drug called Actemra (tocilizumab) for treating patients with active polyarticular juvenile idiopathic arthritis (PJIA) who…
Continue ReadingThe FDA Has Approved a New Form of the Drug Actemra For Polyarticular Juvenile Idiopathic Arthritis
Researchers at Western University and the Lawson Health Research Institute have found that increased water drinking does not slow the decline of kidney function in patients with chronic kidney disease,…
Continue ReadingDrinking More Water May Not Slow Chronic Kidney Disease, Research Suggests
Last week Patient Worthy attended ALD Life’s Community Weekend 2018, where Anne and Joel Buckland from the marketing agency We Do Stories talked about how to bring attention to rare…
Continue ReadingFive Ways You Can Support Rare Disease Charities Through Social Media
The U.S. Food and Drug Administration (FDA) has approved a new use for the treatment Veltassa (patiromer) as an oral suspension (liquid form of the drug) to treat hyperkalemia, a term…
Continue ReadingThe F.D.A. Has Approved a New Label For a Hyperkalemia Treatment
This article contains spoilers The actor-turned-director John Krasinski’s new horror film A Quiet Place has received widespread acclaim, due in part to its representation of deafness. The film is set in post-apocalyptic…
Continue ReadingThe Representation of Deafness in ‘A Quiet Place’
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