Biohaven Pharmaceutical Holding Company Ltd. announced at the end of last month that the company has completed enrollment in its Phase 3 clinical trial of trigriluzole in spinocerebellar ataxia (SCA)…
In April of this year, Argos Therapeutics reported interim results from its Phase 3 ADAPT clinical trial of rocapuldencel-T in combination with the normal standard-of-care for the treatment of newly…
The week before last, innovative biopharmaceutical company DelMar, presented data at the Pediatric Neuro-Oncology Basic and Translational Research Conference from a clinical research with VAL-083 in chemo-resistant glioblastoma patients. Glioblastoma is…
The biopharmaceutical research and development company CytRx Corporation, announced an exciting new update early this month involving the company’s work with aldoxorubicin, a new drug for treatment in soft tissue sarcomas.…
Cystic Fibrosis… For a mother, those words are disheartening, confusing, and so, so frightening. For a child, those words mean countless daily medications, extensive hospital visits, and a shortened life…
Albireo Pharma is in the midst of preparing for an exciting Phase 3 clinical trial to test A4250 for the treatment of progressive familial intrahepatic cholestasis (PFIC). PFIC is a…
Onxeo has recently received the 10th unanimous recommendation from the Data Safety Monitoring Board to continue with the Phase 3 study (coined ReLive) of Livatag®. Livatag® is designed for use…
Xynomic Pharma, a United States-based oncology research and development company, announced at the end of last month that it had entered into a partnership with the University of California San…
The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type C disease. Niemann-Pick disease is a…
Support for Ofev® just keeps coming! Ofev® (nintedanib) was first approved by the U.S. FDA in 2014 for the treatment of idiopathic pulmonary fibrosis (IPF) after clinical trials, especially the INPULSIS…
Results from the Phase 3 CASTOR trial from Janssen Pharmaceutical Companies were recently released, finding that Darzalex (daratumumab) was extremely effective at reducing the risk of multiple myeloma progression or…
Aerie Pharmaceuticals announced exciting efficacy results from the second part of its Phase 3 clinical trial of Roclatan at the end of last month. Roclatan is devised for a group…
Want some more news, events and announcements on hemophilia? We got 'em! Don't forget to share!
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…
What are your thoughts on being a rare disease advocate? Share your stories, thoughts, and hopes with the Patient Worthy community!
© Copyright Patient Worthy
