Note: This patient story was contributed by one of Patient Worthy’s partners, HAE Junior – an organization dedicated to improving the lives of children and young people living with hereditary…
Written by Diane Wilkie My hATTR story began in the mid-1960’s when my father, a healthy, strong, outdoorsy guy in his late 40’s started tripping, experiencing numbness, and having dizzy…
Contributed by Scott Gray. Scott Gray is the co-founder and CEO of Clincierge, a provider of patient support services for clinical trials. Since 2015, Clincierge patient coordinators have managed logistics and reimbursements in…
Introduction I am a 50-year-old woman from the UK who has been suffering with an undiagnosed condition for 11-years for which I am seeking a diagnosis and medical treatment. I…
According to information in a recent press release from Wave Life Sciences, dosing has begun in a clinical trial investigating the RNA editing candidate, WVE-006. The drug treats alpha-1…
For years, scientists have been working on a completely new concept that generates new neurons in mice with Huntington's disease. They were able to demonstrate that the new cells could…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Alicia McGrew’s first baby was born eleven years ago. The new parents checked their baby and found him to be “perfect”. Then Patrick, Alicia’s husband, noticed a few small red…
The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
When Jason Fox was just a child, doctors told his family that he would most likely not live to 18 years old. He had been diagnosed with Duchenne muscular dystrophy…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…
We are excited to announce that Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is…
Having a rare disease can be challenging and isolating. It's often difficult to describe the experience to someone who hasn't gone through it. Mobilizing funds for support, resources, and research…
The parents had brought their baby girl home only four days ago when they received an urgent call from the hospital asking them to bring the baby right back for…
Alzheimer’s disease is a progressive neurodegenerative disease and the most common form of dementia. People with Alzheimer’s disease may have difficulty remembering information, problems with learning, sleep disruptions, poor judgment,…
Amyotrophic lateral sclerosis (ALS), ravages nerve cells in the spinal cord and brain. It is at times called Lou Gehrig’s disease, named after the famous baseball player who died of…
Researchers have demonstrated renewed interest in vaccines that treat Alzheimer’s. The treatments remove toxic proteins from the brain. The author of a recent article in Reuters interviewed ten researchers and…
Zachary Thomas has been an advocate for the mucopolysaccharidosis type I (MPS I) community since he was born. Newborn screening is a public health initiative that tests newborn babies for…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
In total, there are thirteen subtypes of Batten disease. Batten disease, also known as neuronal ceroid lipofuscinosis (NCL), is a rare and inherited neurodegenerative disorder that causes visual impairment, behavioral…
Out of 103,000 people in the United States waiting for a transplant, almost 88,000 individuals on the waiting list need a kidney transplant. These statistics were recently provided through Organ…
It was never just about reaching the summit of Aconcagua, the highest mountain in both the Western and Southern hemispheres. For Scott Osleeb, scaling the heights of this remarkable peak…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
What role do brain alterations play in the onset and progression of spinocerebellar ataxia type 6 (SCA6)? While previous studies highlighted cerebellar changes in individuals with SCA6, the underlying mechanisms…
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