Sometimes when we’re feeling low over the fact that there’s no cure for what ails people with combined variable immune deficiency (CVID), it’s hard to remember that doctors and scientists…
When someone has too much human growth hormone, or GH, which is produced by the pituitary gland, the most frequent outcome is a disorder called acromegaly. It's debilitating, painful, and disfiguring--and…
Happy Friday Patient Worthians! This week we are highlighting a story of a man with cystic fibrosis, racing to breathe and spread awareness of exercise therapy for those with CF.…
If you're like me, you nearly jump out of your skin when somebody makes a loud, unexpected noise nearby. Like the sneaky garbage truck that silently slides up to the…
Imagine, if you will, that you've just been diagnosed with common variable immune deficiency, or CVID. The doctor has answered your most basic questions, and now you're on your way…
In December 2015, the U.S. Food and Drug Administration approved Uptravi (selexipag) for the treatment of pulmonary arterial hypertension (or PAH). Now, others around the world will have the same…
A recent event held in Houston, Texas, raised over $27,000 to help fund research and raise awareness about bone marrow cancer. The Frontier Fiesta 5K Run, now in its third…
Here at Patient Worthy we’ve written before about the heavy toll Severe Combined Immunodeficiency (SCID) can have on families and about the lessons learned from the most well-known SCID case,…
Leí un artículo recientemente por Andrew Smith que me ha desconcertado, no por él, sino más bien los hechos alarmantes que discutí acerca de las personas con fibrosis quística (FQ)…
As families and individuals dealing with rare disease, we all want research and answers sooner- but we soon learn how slow that process is. But check out the hope below: 9500…
One of the many problems with having a rare disease is that very few people know anything about what you, or your loved one, go through on a day-to-day basis.…
Mycosis Fungoides (MF) is a rare form of lymphoma that affects usually affects the skin causing inflammation, tumors, plaques, and redness. It only affects 3 to 4 people in a…
Bernie Mac was a King of Comedy, but he was also living with sarcoidosis. April is sarcoidosis awareness month. What better time to honor one of the kings of comedy with…
He was suffering from the rare myelodysplastic syndrome (MDS) and fell victim to acute leukumia. The former executive director of Uganda Wildlife Authority (UWA), Moses Mapesa passed away on the morning of March…
Measuring Late-night Salivary Cortisol (LNSC) is an alternative to blood and urine for both research and diagnostic use. No wonder patients are opting for salivary cortisol testing over blood and…
OK... I hereby promise to never again make a blonde jokes. Why? Because two blonde sisters, Sam and Alex Kimura, are definitely no joke. They are a powerhouse of promise…
When Myasthenia Gravis is diagnosed in a child, it is called juvenile myasthenia gravis (JMG). But for 4 year old Julius Chatman, the diagnosis was ocular myasthenia gravis. A very rare case,…
Los tratamientos para enfermedades autoinflamatorias comúnmente requieren un paciente para recibir inyecciones debajo de la piel, lo que puede ser difícil si usted es el padre de un niño pequeño…
Life with narcolepsy can be fraught with daily challenges. Excessive daytime sleepiness, cataplexy, or other narcolepsy symptoms can interfere with daily life and activities and make concentration difficult. And for…
Congratulations to Accelmed, the equity investors, who backed SoniVie, a startup company based in Israel that's made a breakthrough treatment device for pulmonary arterial hypertension (PAH). Of course, we owe a…
First of all, what the heck is cataplexy? You may ask if you are unfamiliar the term. Cataplexy is what happens when a person suddenly loses all voluntary muscle control, and…
You may have heard the buzz words "gene therapy" without really understanding what it is, or what's at stake. Gene therapy is when a healthy copy of a gene replaces…
Rare Conect explica la vida de Annie Kwakkel que nació con cistinosis,una enfermedad genética que afecta aproxima a 1 en 100,00 a 200,000 recién nacidos en todo el mundo. Hoy…
Two university professors in Japan are working to make a difference in the lives of people who suffer from narcolepsy and cataplexy associated with narcolepsy. Dr. Hiroshi Nagase and his…
To the casual observer, one-year-old Thor Uran is a happy, healthy toddler with a winning smile and a shock of blond hair fitting for his super-heroic namesake. But Thor’s parents…
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