TSC stands for tuberous sclerosis complex. Often times, it is known as tuberous sclerosis. What is TSC? Tuberous sclerosis complex is a rare genetic disease that can affect multiple areas of…
At the 53rd Annual Meeting of the American Society of Oncology at the beginning of June, Amgen presented promising data from the largest Phase 3 international multiple myeloma trial that…
Passion. Passion doesn’t persist. It is there, and then it is gone. Standing on the diving board is pre-passion. Falling toward the water is post-passion. It's the jump that's ripe with…
Aeglea BioTherapeutics recently provided an update concerning their clinical trial for arginase deficiency disorder. The four-word summary? They still need patients. Aeglea specializes in amino acid metabolism. Their focus is…
ChemoCentrx has been working to develop a drug to combat auto-antibody-associated vasculitis (AAV), C3 glomerulopathy (C3G), and atypical hemolytic uremic syndrome (aHUS). Its name- Avacopan. ChemoCentryx has always had a…
Treatment is expensive. The HealthWell Foundation gets it, and they’re here to help. Insurance companies don’t want to pay more than they have to for your medicine. It’s the nature…
Lysogene, a biopharmaceutical company, announced at the end of May that enrollment in SAMOS (Sanfilippo A Multinational Observational Study) was completed. This is extremely exciting news for Sanfilippo syndrome (MPS…
Happy Father's Day Weekend Patient Worthians! This week we have an amazing essay written by CMT Warrior Sarah Magno regarding her personal experience overcoming rare disease obstacles. We also have some…
Want some more news, events and announcements on IPF? We got 'em! [one_half] [/one_half] [one_half_last] Miracle Drug to Become Much More Affordable Soon IPF Treatment Developments You Should…
Come mid-January, eager and anxious girls flood the roads connecting all of the sororities at my school for recruitment, in perhaps what is the most important week of their first…
On October 20th and 21st, the 2017 Rare Neuro-Immune Disorders Symposium (RNDS) will be held in Columbus, Ohio. The event is meant to bring together patients, families, researchers, and all…
I’ll admit that I ran wild as a kid. I never got into trouble—you know what I mean, I was never arrested, no animals were harmed, and no injuries were…
In late May of this year, Aurinia Pharmaceuticals Inc. dosed its first patient with voclosporin in a Phase 3 trial known as AURORA. AURORA is a confirmatory clinical trial to…
Everybody wants to win. Winning gives a certain pride and joy that keeps us wanting more. Winning becomes a motivating factor for a person to do her best so she…
Sarah Trask had childhood cancer 32 years ago and had four brain surgeries. Sarah is a survivor. As a result, epilepsy is the side effect she is living with. Sometimes…
More exciting news from the Annual American Society of Clinical Oncology Meeting last week! Onconova Therapeutics announced results from a Phase 2 study of oral rigosertib as a treatment for…
Once upon a time there were three young brothers, and SPOILER ALERT- this part of the story, unfortunately, does not have a happy ending. Each brother suffered: Each had chronic…
Hey, do you guys remember that Mark Wahlberg movie that came out not long ago? You know, the one where he’s a cop? No no, not that one. The serious…
The term “superfood” is thrown around a lot. It is used for everything from salmon to spinach, from pistachios to pumpkin, from chia seeds to quinoa. When people use the…
"You're so full of sh*t." We've all heard it or said it at some point, be honest! But for one 22 year-old man in China, he was literally full of…
In April of this year, Rigel Pharmaceuticals, Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. FDA for fostamatinib, a drug for patients with chronic…
With all the new products, widgets, and gadgets out there meant to clutter our life, there is one that manages to maintain style, grace, and cool points while providing a…
Attention hereditary angioedema patients: preventative treatment is on the horizon! Shire plc is one of the global leaders in rare disease research and pharmacology, and the group recently released positive…
My freshman year of high school, I began to get strange, red spots on my body. They initially developed on my arms, but within a couple of days, they had…
Want some more news, events and announcements on Batten disease? We got 'em! [one_half] [/one_half] [one_half_last] Pioneering Brain Surgery May Give This Toddler More Time FDA Press Release…
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