Maroteaux-Lamy Syndrome, also known as mucopolysaccharidosis (MPSVI), is a lysosomal storage disorder. It's one of a number of disorders that affects specific enzymes in specific locations. The job of these…
Hey Patient Worthy Community! Did you you know that September is Rheumatic Disease Awareness Month (RDAM for short!)? Well, now you do! Don't feel bad if you didn't know. This…
I shared it with my family. I shared it with my wife, my mother and my brother of course, and they were grateful that it wasn’t something that was going to…
Imagine you're a new parent and your two-month-old starts experiencing some bizarre symptoms. Along with his high fever, his skin turns red and starts peeling at an alarming rate. Multiple infections follow;…
Cistinosis, una de las muchas enfermedades raras, genéticas en el mundo de hoy ... Como en cualquier buen artículo, el lector primero debe estar provisto de una breve historia del…
En un comunicado de prensa informó de BusinessWire, la Fundación de Investigación cistinosis, CRF, concedido subvenciones sin precedentes de $ 1.022.879 a instituciones de investigación por lo que es posible…
Ahh, el milagro de la vida. Aunque es una cosa hermosa, el embarazo puede convertirse en una cosa cada vez más doloroso para las personas con artritis y enfermedades relacionadas.…
Happy Friday Patient Worthians! PW Contributor Nolan is back in our Video Series with his details on dystonia treatment. We also have some cool info on EDS gadgets! What do pharmaceutical companies mean…
Life is hard. Life gets even harder for a woman once a month. The topic of menstruation always has been sort of taboo. I remember getting my first period. While crying on the toilet,…
If you have dopamine beta hydroxylase deficiency (DBHD), you're part of a very exclusive club, one that you probably would have passed on if you'd had the chance. With this…
Lifestyle adjustments. You know your life will change once you get a disease diagnosis. These changes may be easy or hard depending on how you are currently living life. For…
Imagínese estar preocupado porque su risitas 6 meses de edad hijo no quiere comer, tiene estreñimiento, y la pérdida drástica de peso. Los médicos le dicen que tiene una enfermedad…
Remember the Parkinson’s disease Ice Bucket Challenge mania that spread across the globe? Did you find yourself overflowing with ideas about equally awesome challenges or ways to raise awareness and…
Dysautonomia is an umbrella term for a number of disorders of the autonomic nervous system (ANS). But to understand the collection of conditions called "dysautonomia," first we have to understand…
As a self-described science nerd, I always feel a thrill when I read about a new drug reaching Phase 3 of a clinical trial. At that point, chances are very…
Being a caregiver is among the most important jobs a person can take on, but it comes with its own set of perils: isolation depression sleep deprivation So, if you…
Gran yerba! Parche de marihuana en camino ... Y No estamos soplando humo. Zynerba Pharmaceuticals está trabajando hacia parches para entregar los productos químicos derivados del cannabis directamente al torrente…
There [are] treatment[s] for symptoms for dystonia. For neuropathy they can give you things like anti-depressants which have been known to curb neuropathy. They can do botox injections into the muscles…
Imagine having a student in class that looks... a bit like this on a regular basis. You may have a lot of thoughts about what might be going on. The…
Myasthenia gravis (MG) is a neuromuscular condition that causes weakening of the body's skeletal muscles. It's a rare disorder and there is no cure at this time, but research continues and…
Doggone, it feels good to fight amyloidosis! The condition causes abnormal proteins to be deposited in organs like the kidneys and heart. That's got bad news written all over it. But…
Mientras que el verano vientos hacia abajo, muchos de nosotros están luchando para conseguir una vacacion más. Ya se trate de un día en la playa, un picnic en el…
Hey, do you like helping people--especially people who have amyloidosis? Also, do you live in or near New Jersey? Or will you be in the area later in September? If…
Kallmann syndrome is a genetic condition caused by hypogonadotropic hypogonadism (HH), of which four separate types have been identified. People with Kallmann syndrome experience olfactory deficiencies, and puberty that is either…
Sjögren’s syndrome is an autoimmune disease that affects the moisture-producing glands in the body, including the salivary and tear glands. Very often, it's accompanied by rheumatoid arthritis. It's thought to…
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