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…
Orkambi (ivacaftor / lumacaftor) ist die zweite Präzisionsmedizin, die für den Einsatz in Großbritannien von Menschen mit zystischer Fibrose zugelassen wird. Der Fang? Es wird derzeit nicht von der National…
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…
“If you half-ass something, you should only expect to get half an ass back.” This was the sort of lesson I learned from my dad. The moral is there, even…
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…
Orkambi (ivacaftor / lumacaftor) es la segunda medicina de precisión con licencia para uso en el Reino Unido por personas con fibrosis quística. ¿La captura? Actualmente no es proporcionado por…
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…
Orkambi (ivacaftor / lumacaftor) è la seconda medicina di precisione da licenza per uso nel Regno Unito da persone con fibrosi cistica. La presa? Non è attualmente fornito dal Servizio…
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…
Orkambi (ivacaftor / lumacaftor) est le second médicament de précision à être autorisé à être utilisé au Royaume-Uni par des personnes atteintes de fibrose kystique. La prise? Il n'est actuellement…
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,…
It’s more difficult to prove something to someone than we often imagine. Then again, there are also times when proving something that may or may not exist is relatively easy.…
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…
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