Imaginez vous asseoir à la table. Vous mettez une quantité normale de nourriture sur votre assiette et vous commencez à manger. Ensuite, vous prenez une deuxième aide de tout et…
If you were to rate the severity of different types of epilepsy from one to 10, 10 being the most severe, Dravet syndrome would be an 11. Dravet is catastrophic…
At a recent conference, Jazz Pharmaceuticals presented positive efficacy results from the JZP-110 (TONES) studies in adult patients with sleep impairment conditions and the excessive sleepiness associated with these diseases.…
For people living with Idiopathic Pulmonary Fibrosis (IPF), every day is part of an ongoing battle to hold on tight to lung function as long as possible. That battle can…
I came across an interesting blog about World Hemophilia Day (WHD) which was April 17th, 2017. It’s an interesting read, and fairly simple, one every woman should know about for inspiration,…
Rhythm Pharmaceuticals is currently developing a melanocrotin-4 (MC4) receptor therapy called setmelanotide for the treatment of obesity and related disorders caused by genetic defects. This therapy could potentially be used to…
At the recent Annual American Society of Clinical Oncology (ASCO) Meeting, bluebird bio, Inc. released updated data from its Phase 1 trial of bb2121. bb2121 is a new anti-BCMA chimeric…
The highly-anticipated data from Pfizer's Phase 3 Octave Studies of tofacitinib in ulcerative colitis patients were published early last month in The New England Journal of Medicine. The data overall…
BioLineRx Ltd. announced in early May that following a successful meeting with the U.S. FDA, the initiation of a Phase 3 clinical trial for stem cell mobilization in multiple myeloma…
Celgene Corporation, partnered with Acceleron Pharma Inc., announced at the beginning of this month that target enrollment of myelodysplastic syndromes (MDS) and beta thalassemia patients for the MEDALIST and BELIEVE…
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…
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…
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…
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