Adrenoleukodystrophy (ALD) is a genetic disorder that is very rare and usually occurs in childhood. People with ALD have two options for treatment, both of them less than favorable and…
We have reported a lot on the amazing possibilities that stem cell treatment offer the rare disease community. But something we assume, though we don't hear much about, is that…
Patient Worthy attended the National Hemophilia Foundation's 69th Annual meeting in Chicago and we are thrilled to report on some of the exciting new developments for patients with bleeding disorders.…
Bilateral deep brain stimulation (DBS) has been known to help with symptoms of dystonia. But for a form of tardive dyskinesia known as tardive dystonia, DBS hasn't been studied as…
The Myositis Association (TMA) supports the myositis community, helps to fund myositis research and increases myositis awareness. Registration is currently OPEN for their 2017 Annual Patient Conference. Details below: Who: The…
In a study supported by the National Institute of Health (NIH), researchers at Iowa State University found that a drug called A15/283 helped to ameliorate symptoms of a mild form…
The heart-breaking story of baby Charlie Gard, who recently died of a rare genetic condition called encephalomyopathic mitochondrial DNA depletion syndrome, touched the lives of families around the world. Although…
This month, Soliris®, a treatment produced by Alexion Pharmaceuticals, Inc. was approved by the European Commission for the treatment of Refractory Generalized Myasthenia Gravis (gMG). Myasthenia gravis (MG) is a…
If you're looking for a time table for adjusting to, and accepting, the diagnosis of a rare disease, you won't find one, because there isn't one. The variety of rare…
Duchenne muscular dystrophy (DMD) is a heartbreaker for the the 250,000 families worldwide whose sons are born with this debilitating, progressive disease. It is rare, and yet the ripples of its…
Cancer. This word can inspire fear in even the most stalwart of individuals. How many other diseases are known by a phrase involving just the first letter? The Big C.…
The first Disorder: The Rare Disease Film Festival (RDFF) is coming to Boston, MA this October! Check out details below: Who: Rare Outreach Coalition, LLC. What: Disorder: The Rare Disease Film…
At 54 years of age, Kathleen was healthy and physically fit. She knew about her grandfather’s death at the age of 30 to a heart attack. She also knew about…
Diamond Blackfan anemia (DBA) occurs when the bone marrow in one’s body either does not produce red blood cells or doesn't create enough of them. Sometimes called Blackfan Diamond anemia,…
Happy Back-to-School Week Patient Worthians! If you're going back to school or your children are going back to school, we know it's a hectic and fun time of year. So…
When I was a kid, pedal power was the way to go. In fact, it was the only way to go—anywhere. It was a mode of transportation for us. We…
In recognition of Spinal Muscular Atrophy Awareness Month, Joe Akmakjian, an SMA warrior/blogger, is promoting a unique understanding of his rare disedsase. Originally published on the Muscular Dystrophy Association's Strongly…
Britain may be making its Brexit, but people with rare diseases may want to keep a close eye on the European Union (EU) and its newly formed European Reference Networks (ERNs).…
Mended Hearts is a nonprofit group that helps bring hope to heart disease patients, caregivers and families. In Alabama specifically, the local Mended Hearts group had not been active for…
One night in March 2016,13-year-old Treyvon Summers experienced extreme pain that woke him up out his sleep. This was the start of him losing his mobility in both his legs.…
Stepping on a Lego—that piercing pain is familiar to just about everyone, and it hurts an unreasonable amount. You step on other things all the time, but Legos seem to…
Whenever I feel the stress of life getting to me, I hop over to YouTube and search “random acts of kindness” or “heartwarming acts.” Some of the results? The police…
Sickle cell disease, hemophilia, and thalassemia are a few of the inherited blood disorders. Dr. Waveney Charles is a hematologist, or specialist in blood-related conditions. She believes that the number…
In my home, everything is shared with our dogs. When eggs are made for breakfast, the dog’s eggs are served scrambled. When it’s time to exercise, we all head outside…
Loooong before myasthenia gravis (MG) came into the picture, Cathie’s mom used to joke that Cathie must be part fish. She was always in the water. It started with swimming…
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