At the 2017 Global Genes Advocacy Summit, one of the break-out sessions discusses the drug development process. But it goes beyond this to educate us, the patients, on how imperative…
We have written about the anatomy of an HAE attack before. It’s a living nightmare. HAE is a rare genetic disorder. It is characterized by recurring, unexplained, severe swelling underneath…
Patient Worthy is fortunate enough to be at the 2017 Global Genes Rare Patient Advocacy Summit this week. While the personal stories that have been shared are highlights of the…
Parkinson’s disease is a degenerative movement disorder of the central nervous system that insidiously forms, even starting subtly with a single tremor, progressing into slurred speech, loss of balance, and…
When Alexander Fleming used penicillin from a fungus to fight bacterial infections, he knew he was onto something that would revolutionize the world of medicine. Now, a modern-day “Fleming” is…
Everyone seems to have Epstein-Barr in my family. My mom, who has celiac disease and sciatica, and my sister, who has lichen sclerosis. Given that typical MDs don't seem to have…
Norway scientists Suchi Mittal, et al. have found compounds that reduce levels of a key gene that increases the risk of developing Parkinson's disease. Parkinson's disease is a degenerative central…
A record amount of dollars is heading to help those fighting Waldenstrom’s macroglobulinemia (WM). According to its website, for the first time, the International Waldenstrom’s Macroglobulinemia Foundation (IWMF) approved $2.2…
Duchenne muscular dystrophy is most common in males, and is characterized by muscle weakness that progresses over time. Onset is as early as three years of age and the life…
Mantle Cell Lymphoma (MCL) is a very rare cancer of the blood. So rare in fact, that there are only 4,200 cases diagnosed in the U.S. each year and…
The human brain is a mystery. Although enormous strides have been made to understand how it works and how our neuro pathways respond to various stimuli, there is still so…
When I first read this story in Fortune about the Zika Virus potentially being used to treat a rare form of deadly brain cancer called glioblastoma, I immediately thought of…
The Indiana Hemophilia & Thrombosis Center in Indianapolis has long been a leader in innovative patient care. They teamed up with MicroHealth, a company founded by hemophilia patient Aaron Craig…
An easier diagnosis may be coming for ulcerative colitis patients, as researchers at Georgia State University recently used a special type of spectroscopy to examine the blood serum of mice…
Researchers at The Ohio State University Wexner Medical Center are curious about how nicotine may effect patients battling sarcoidosis. Sarcoidosis effects everyone differently. It occurs from excess granulomas, often resulting in…
A team of researchers at the University College of London (UCL) looking at the genome of childhood-onset dystonia patients has stumbled on a startling discovery: 28 of the patients had…
Batten disease, also known as neuronal ceroid lipofuscinoses (NCLs) is a fatal, inherited disorder of the nervous system. It involves a build up of lipopigments that accumulate and kill brain…
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…
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…
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…
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…
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).…
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…
A recent survey of more than 150 idiopathic pulmonary fibrosis (IPF) patients demonstrates the invisible burden the rare disease causes. It is no secret that any rare disease has the…
Over the years, I've talked with dozens of people with Parkinson's disease (PD). Male or female, old or young, they tend to have one characteristic in common, even if they…
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