Rare Disease Meme – Coffee: A cup with of benefits
Congratulate this Mom- She knew something was wrong. She went through 19 specialists in 4 years. She eventually got a diagnoses. Her son, had Syngap1, a very rare, gene based,…
Congratulations to Kristin, the winner of a $500 travel stipend to get to the 2016 Acromegaly Community Bi-annual Meeting! Patient Worthy is so excited to meet you Kristin! We are excited to…
It's officially April and it's officially the WEEKEND! PW Contributor Alexis writes of how her experience with PTSD relates to going through the process of diagnosis and treatment of chronic…
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…
World PI Week is coming up later this month, from April 22-29. So what better way to celebrate than to learn what exactly PI is! Not to be confused with Pi…
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,…
If there was one song that could get me poppin’ throughout my childhood, it would have to be “Parents Just Don’t Understand” by the Fresh Prince himself. Come on, you…
we may look different on the outside, but each one of us is unique on the inside, and that holds true more so when it comes to treatment options. I'm…
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…
Do you have narcolepsy? Have you ever heard any of the following? “You should get a little more rest; then you’ll be fresh and ready to go.” “Exercise and eating healthy…
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…
Discovery Phase Drugs for Orphan Eye Diseases IVMED-100- 80- 90 are currently without generic drug names. They are under development for the treatment of orphan eye diseases by iVeena, LLC who has 6…
RESEARCHERS BELIEVE SARCOIDOSIS IS ASSOCIATED WITH AN ABNORMAL IMMUNE SYSTEM RESPONSE, BUT WHAT TRIGGERS THIS RESPONSE IS NOT KNOWN. NOR DO DOCTORS KNOW WHETHER HEREDITY, ENVIRONMENT, OR LIFESTYLE AFFECTS THE…
Vitae Pharmaceuticals Announces Positive Top-Line Results From Initial Phase 1 Study of First-in-Class RORyt Inhibitor VTP-43742 in Autoimmune Disorders back in September. Vitae Pharmaceuticals, Inc. (“Vitae”) is developing VTP-43742 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…
¿Y ahora qué? Llegaste al doctor para hacerte un chequeo y sales con un diagnostico que cambia tu vida entera. Sea, Distonía, Acromegalia, Angioedema Hereditario (AEH) o la enfermedad de…