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BridgeBio Pharma and Affiliate Origin Biosciences Announce FDA Approval of NULIBRY™ (fosdenopterin), the First and Only Approved Therapy to Reduce the Risk of Mortality in Patients with MoCD Type A


BridgeBio Pharma and Affiliate Origin Biosciences Announce FDA Approval of NULIBRY™ (fosdenopterin), the First and Only Approved Therapy to Reduce the Risk of Mortality in Patients with MoCD Type A
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Molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that progresses rapidly, results in severe and largely irreversible neurological injury, and has a high infant mortality rate; median overall survival age is about four years
Clinical trials with NULIBRY or recombinant cPMP showed a meaningful increase in overall survival compared to a natural history study
NULIBRY is BridgeBio’s first FDA-approved therapeutic
PALO ALTO, Calif., Feb. 28, 2021 (GLOBE NEWSWIRE) BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio) and affiliate Origin Biosciences, Inc. (Origin) today announced the U.S. Food and Drug Administration (FDA) has approved NULIBRY™ (fosdenopterin) for Injection as the first therapy t ....

Bridgebio Pharma , Natural History Study , Molybdenum Cofactor Deficiency , Origin Biosciences , Rare Diseases , இயற்கை வரலாறு படிப்பு , ஆரிஜிந் உயிர் அறிவியல் , ரேர் நோய்கள் ,

New Multi-institutional Study Analyzes Tumor Tissue and Patient Data to Improve Care


Credit: NCI-CONNECT Staff
Studying tumor tissue and clinical data across our national network of institutions will help researchers advance their knowledge of rare brain and spine tumors faster.  
NCI-CONNECT is launching a study across its network of clinical collaborators to better understand rare brain and spine tumors. The study collects tumor tissue and comprehensive clinical data from deceased patients with rare brain and spine tumors and people that were pregnant at diagnosis or became pregnant after diagnosis of a primary brain tumor.
“The data are out there waiting to be analyzed and understood. The more data we collect, the better we can design clinical trials and study therapeutic options that improve the outcomes of patients,” says Marta Penas-Prado, M.D., principal investigator of the study and an associate research physician in the Neuro-Oncology Branch, Center for Cancer Research, NCI, NIH. ....

Kenneth Aldape , Shlomit Yust Katz , Marta Penas Prado , Cancer Research , Laboratory Of Pathology , Rabin Medical Center , Oncology Branch , Neuro Oncology Branch , Tissue Outcomes Study , Pregnancy Sub Study , Natural History Study , Brain Tumor Trials Collaborative , மார்த்தா ஆண்குறி ப்ராடோ , புற்றுநோய் ஆராய்ச்சி , ஆய்வகம் ஆஃப் நோயியல் , ராபின் மருத்துவ மையம் , புற்றுநோயியல் கிளை , நரம்பியல் புற்றுநோயியல் கிளை , திசு ஔட்கம்ஸ் படிப்பு , ப்ரெக்நெந்ஸீ துணை படிப்பு , இயற்கை வரலாறு படிப்பு , மூளை கட்டி சோதனைகள் கூட்டு ,