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…
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