According to a story from the National Center for Advancing Translational Sciences, a group of scientists from the National Institutes of Health (NIH) has unearthed a new mechanism that could…
A team of childhood cancer researchers, known as the Pediatric Cancer Dream Team, were first created in 2013 through collaboration between the charitable organizations Stand Up To Cancer (SU2C)…
So what is Fabry disease you ask? You came to the right place! Fabry disease is a genetic, lysosomal storage disorder that occurs when a specific kind of fat known…
Have you read part one of Loukisha's story? Check it out here! When I imagined life with my husband and children my plans didn’t include surgeries, walkers, canes, a wheelchair,…
The US Food and Drug Administration have just approved a genetically engineered swine research model for the rare disease ataxia telangiectasia for commercial use. The model, officially named the ExeGen…
This week is National Infant Immunization Week! For many of us, it was an anxiety-riddled memory of getting a ton of shots at the doctor's office when we were younger. And…
According to a story from PR Newswire, a recent study indicated that researchers that were focused on kidney illnesses such as chronic kidney disease, congenital nephrotic syndrome, and glomerulonephritis have not been…
According to a story from The Motley Fool, The gene therapy development company bluebird bio has been working on a treatment for beta thalassemia, and it has the potential to…
From birth until my mid-thirties I was a very active person. I loved dancing West Coast Swing, doing yoga, and cooking large meals for my ‘Ohana. The word "'Ohana" describes…
Today is International Fibrodysplasia Ossificans Progressiva (FOP) Awareness Day! We at Patient Worthy understand that a lot of these diseases you may not have heard of - which is exactly why awareness…
Gabriella is a happy young girl and student. She enjoys school and especially loves her math class. She loves animals and has several pets that she lovingly cares for. Despite…
Happy Friday Everybody! This week we're spotlighting three patient stories: an ALD patient full of optimism and love, a beauty pageant contestant with a noble goal, and a woman with…
According to a story from biotechconnectionbay.org, medical researchers are beginning to more closely investigate that possibilities that drug repurposing, or using old drugs for treating different illnesses, could offer. While…
According to a story from statnews.com, a drug derived from substances originating in the cannabis plant may be nearing approval by the U.S. Food and Drug Administration. The drug is…
For many, being diagnosed with any disease – let alone a rare disease – is understandably devastating. Many have described managing their new lives as learning a new language or…
Researchers are arguing that the certain drugs, including the erectile dysfunction drug Viagra, could be used as cancer treatments, reports MedicalXpress. This theory is based on a range of pre-clinical…
A recent study, published in Nature Communications and reported on by the BBC, has discovered five genes that are linked to pulmonary arterial hypertension. Pulmonary arterial hypertension (PAH) is the…
The 2018 Mrs. Maine International Pageant will be so much more than a celebration of poise and beauty. It will also make history when Devan Demmons will proudly walk on…
The pharmaceutical company Lonza has just opened a new state-of-the-art manufacturing facility to produce cell and gene therapies. You can find the full press release here, on Lonza’s website.…
According to a story from Summit Medical Group, a recent study indicates that survivors of testicular cancer may have a greater risk for heart disease. Compared to many types of…
According to a story from stv.tv, 14-year-old Kira Noble has been fighting against neuroblastoma for around over four years. She first began to realize that something was wrong when her…
According to a story from wired.com, a new genomics marketplace called Helix will soon start selling genetic tests directly to consumers. These tests are the same ones that doctors use,…
According to a story from globenewswire.com, the pharmaceutical companies Ultragenyx and Kyowa Kirin recently issued an announcement that the U.S. FDA has granted approval to their new treatment Crysvita (burosumab-twza).…
According to a story from news.vanderbilt.edu, Charlotte Haffner has been working hard to advocating on behalf of amyloidosis patients. She recently underwent a heart transplant and stem cell transplant in…
Researchers have found that forms of certain genes can influence how successful a blood and marrow transplant is, reports Roswell Park. This has important implications for understanding patient outcomes and…
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