Want some more news, events and announcements on hemophilia? We got 'em! [one_half] [/one_half] [one_half_last] FDA Clears New Drug for Hemophilia B Treatment Virtual-reality Game Helps This Boy…
Fino a poco tempo fa, il termine ROHHAD non significava nulla. Ma poi ho sentito parlare di un bambino canadese di 11 anni chiamato Denika Bailey che ha questo raro…
L'emiplegia si verifica normalmente durante la gravidanza, o molto rapidamente dopo la nascita. I genitori sono fortunati ad avere alcuni momenti con il loro bambino prima che questa condizione cambia…
L'hémiplégie se produit normalement pendant la grossesse, ou très rapidement après la naissance. Les parents ont de la chance d'avoir quelques instants avec leur enfant avant que cette condition ne…
Je suis sûr que vous avez entendu parler de la SLA après que le «Défi du dégel de glace» ait balayé la nation en 2014, mais peu ont probablement pris…
Sono sicuro che hai sentito parlare di ALS dopo che la "Ice Bucket Challenge" ha spazzato la nazione nel 2014, ma pochi ancora probabilmente hanno preso il tempo per ricercare…
On a beautiful summer day, a baby girl named Remi made her debut in the world. To all appearances, everything was normal—10 fingers, 10 toes, and bright eyes. Her parents…
Imagine being told that your son probably wouldn’t live beyond his twenties when he was only six years old. Unfathomable. But that’s the news the Vella family received and had…
Immagina di sedersi al tavolo. Metti una normale quantità di cibo sul piatto e cominci a mangiare. Poi, prendi un secondo per aiutare tutto e tu mangi anche quello. La…
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…
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