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…
Andrew Macnab received a diagnosis of dermatomyositis last year. His sister Helen Keith is preparing for her first marathon in order to help Andrew. The money Helen raises will go…
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…
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…
According to a story from Science Daily, a recent study found that a rigorous exercise program reduced fatigue in testicular cancer patients and helped boost their self-esteem. Patients with the…
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…
For a long time, researchers thought that chronic liver inflammation was the driving force of cancer, because the inflammation allows the cancer cells to divide, preventing them from dying. But…
According to a story from Cambridge News, Ethan Monk's Williams syndrome managed to evade detection until just two years ago. Ethan had some peculiar symptoms, such as delayed development, unusual…
According to a story from globenewswire.com, the drug development company Galectin Therapeutics, Inc., recently announced its plans to go forward with Phase 3 trials for GR-MD-02. The company is dedicated…
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…
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.…
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…
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…
According to a story from Financial Buzz, the pharmaceutical company Albireo Pharma recently announced that the first patient has been enrolled in its Phase 3 trial testing its experimental candidate…
How far would you go to stop cancer? Not metaphorically. Literally. Caryn Lubetsky has an answer. It’s the distance between Key Largo and Key west. Caryn Lubetsky is an attorney,…
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…
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…
According to a story from news-medical.net, researchers working with the Institute for Experimental Pediatric Endocrinology of the Charité - Universitätsmedizin Berlin were able to successfully treat young patients that were obese…
As someone who has suffered with chronic vertigo for the past year and a half, and who deals with a chronic vestibular condition, I know all too well how debilitating…
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,…
The hemoglobin A1C test is a test that is commonly used to determine if patients have diabetes. A study last year in February 2017, by the American Medical Association found…
A woman from the UK, whose husband was diagnosed with Parkinson's disease many years ago, has the unique distinction of being able to detect the disease in patients through a…
New research is showing that patients sometimes use crowdfund websites to pay for fake stem cell transplants and other dubious medical procedures, according to a story from medicalxpress.com. Crowdfunding websites…
An international team of researchers studying the effects of progressive familial intrahepatic cholestasis (PFIC) in children have found that half of the children had received a liver transplant by age…
A rare but serious disease caused by flesh-eating bacteria has been studied, and researchers have identified the mechanism by which it progresses. This has lead them towards potentially effective treatments…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
