The inaugural Homocystinuria Network America Conference was held April 21st-22nd in Westford, MA. The families in attendance traveled from all across the US for the event. They all have different…
To read part one of this story, click here. Morgan is a young woman in her early twenties who has been living with symptoms of extreme pain on a daily…
According to a story from People, a little girl from Ohio has faced major health challenges since her birth. Grace Rosian, who was born with Down syndrome, was diagnosed with…
The US Food and Drug Administration (FDA) has just awarded Breakthrough Therapy Designation to a drug (OMS721) being developed to treat thrombotic microangiopathy that has developed as a result of haematopoietic stem…
At the age of 28, Suzy remembers medical students feeling her puffy hands and feet. Her feet were so expanded she had to wear wide shoe sizes. Her fingers were…
When I was diagnosed with Parkinson's Disease (PD) in 2000, I first thought of my three children and how my diagnosis would impact them. How would I tell them? Would I…
Imagine not being able to eat because you cannot swallow your food. Add to that intolerable pain and the inability to sleep at night. These are some of the symptoms…
In order to honor Cystinosis Awareness Day, which takes place on May 7th each year, a man will attempt to walk 57 miles in a single day in order to…
A new device to treat incontinence is being developed by Peter Sayet at Precision Medical Devices Inc., reports the Orlando Sentinel. Incontinence is the term used when people pass urine…
Happy Friday Everybody! This week we're spotlighting two parts of one patient story. We also have news on Alfie Evans and epilepsy research. Sit back and enjoy this week's Editor's…
Researchers at the University of Hong Kong have discovered that a drug used to treat muscular dystrophy also works on a hereditary heart disease called Lamin A/C-related dilated cardiomyopathy. The…
A group of researchers led by Professor Gholson Lyon has discovered a genetic mutation that is linked to certain traits, reports Medical Xpress. The method used to discover it was…
The prolonged legal battle over Alfie Evans, a toddler with a serious undiagnosed degenerative neurological disease, has reached a final verdict. The UK doctors currently caring for Alfie made the…
According to a story from edinburghnews.com, mother Karen Gray is about to seek access to cannabidiol oil for her five-year-old son, Murray, who has a rare type of epilepsy, Myoclonic Astatic…
According to a story from tapinto.net, the Merrill family was crushed upon learning that their five-year-old daughter Leila was born with Sanfilippo syndrome, a lysosomal storage disease that ultimately causes…
According to a story from Cosmos Magazine, Dr. Kathryn North is committed to researching the vast expanse of the human genome in order to pinpoint the ways in which genes…
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…
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