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…
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.…
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…
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…
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…
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…
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…
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…
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…
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…
GW Pharmaceuticals is finding out... Epidiolex® (manufactured and tested by GW Pharmaceuticals) is a leading medical cannabinoid candidate for the treatment of rare, resistant, and early-onset epilepsy syndromes such as…
In September, 2015, the U.S. FDA approved NUWIQ®, a recombinant antihemophilic factor (protein essential for normal blood clothing) developed by Octapharma AG, for the treatment of hemophilia A in children…
Want some more news, events and announcements on Fabry disease? We got 'em! [one_half] [/one_half] [one_half_last] This Fabry Disease Treatment Has Had a Rocky Journey Regulatory Pathway Announced…
Want some more news, events and announcements on Gaucher disease? We got 'em! [one_half] [/one_half] [one_half_last] Promising Molecule Identified as Possible Treatment for Gaucher, Parkinson's Diseases NICE Changes Mind…
Want some more news, events and announcements on Dravet syndrome? We got 'em! [one_half] [/one_half] [one_half_last] Opko Health Inc. is Closing In on a Treatment for Dravet Syndrome…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
