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nulibry

a precursor to molybdenum cofactor; structure in first source
Also Known As:
ALXN1101; cPMP cpd; cyclic pyranopterin monophosphate; fosdenopterin
Networked: 8 relevant articles (0 outcomes, 1 trials/studies)

Bio-Agent Context: Research Results

Experts

1. Christodoulou, John: 2 articles (11/2015 - 09/2014)
2. Veldman, Alex: 2 articles (11/2015 - 05/2010)
3. Kang, Connie: 1 article (06/2021)
4. Adeniyi, Oluseyi: 1 article (01/2021)
5. Donohue, Kathleen: 1 article (01/2021)
6. Farrell, Sheila: 1 article (01/2021)
7. Filie, Jane: 1 article (01/2021)
8. Hager, Rebecca: 1 article (01/2021)
9. Joffe, Hylton: 1 article (01/2021)
10. Karp, Jacqueline: 1 article (01/2021)

Related Diseases

1. Molybdenum cofactor deficiency
06/01/2021 - "Fosdenopterin (NulibryTM) is a synthetic cyclic pyranopterin monophosphate that is being developed by Origin Biosciences (a subsidiary of BridgeBio Pharma) for the treatment of molybdenum cofactor deficiency (MoCD) type A. "
01/01/2020 - "rWGS revealed compound heterozygous variants in the molybdenum cofactor synthesis gene, type 1A (MOCS1 c.*7 + 5G > A and c.377G > A); a provisional diagnosis of molybdenum cofactor deficiency on day of life 4. An emergency investigational new drug application for intravenous replacement of the MOCS1 product, cyclic pyranopterin monophosphate, was considered, but felt unsuitable in light of the severity of disease and delay in the start of treatment. "
09/01/2014 - "At present, treatments for the clinically significant defects of the purine pathway are restricted: purine 5'-nucleotidase deficiency with uridine; familial juvenile hyperuricaemic nephropathy (FJHN), adenine phosphoribosyl transferase (APRT) deficiency, hypoxanthine phosphoribosyl transferase (HPRT) deficiency and phosphoribosyl-pyrophosphate synthetase superactivity (PRPS) with allopurinol; adenosine deaminase (ADA) and purine nucleoside phosphorylase (PNP) deficiencies have been treated by bone marrow transplantation (BMT), and ADA deficiency with enzyme replacement with polyethylene glycol (PEG)-ADA, or erythrocyte-encapsulated ADA; myeloadenylate deaminase (MADA) and adenylosuccinate lyase (ADSL) deficiencies have had trials of oral ribose; PRPS, HPRT and adenosine kinase (ADK) deficiencies with S-adenosylmethionine; and molybdenum cofactor deficiency of complementation group A (MOCODA) with cyclic pyranopterin monophosphate (cPMP). "
2. Severe combined immunodeficiency due to adenosine deaminase deficiency
3. Complementation Group A Molybdenum Cofactor Deficiency
4. Brain Diseases (Brain Disorder)
5. Body Weight (Weight, Body)

Related Drugs and Biologics

1. Hypoxanthine
2. Uridine
3. Transferases
4. S-Adenosylmethionine (Ademetionine)
5. Ribose-Phosphate Pyrophosphokinase (Phosphoribosyl Pyrophosphate Synthetase)
6. Ribose (D-Ribose)
7. Purine-Nucleoside Phosphorylase
8. Polyethylene Glycols (Polyethylene Glycol)
9. Hypoxanthine Phosphoribosyltransferase (Hypoxanthine Guanine Phosphoribosyltransferase)
10. Enzymes

Related Therapies and Procedures

1. Bone Marrow Transplantation (Transplantation, Bone Marrow)
2. Therapeutics
3. Intravenous Administration