Rare Disease and Orphan Drug Development
There are 7,000 rare diseases impacting 350 million patients worldwide, yet only 300 of these…
CertaraAugust 3, 2016
Population Pharmacokinetic and Pharmacodynamic Analyses from a 4-Month Intra-dose Escalation and Its Subsequent 12-month Dose Titration Studies for a Human Monoclonal Anti-FGF23 Antibody (KRN23) in Adults with X-linked Hypophosphatemia
X-linked hypophosphatemia (XLH) is an inherited metabolic bone disease with abnormally elevated serum FGF23 resulting…
CertaraApril 1, 2016
Chemotherapy of Second Stage Human African Trypanosomiasis: Comparison Between the Parenteral Diamidine DB829 and Its Oral Prodrug DB868 in Vervet Monkeys
Human African trypanosomiasis (HAT, sleeping sickness) ranks among the most neglected tropical diseases based on…
CertaraFebruary 15, 2015
Ammonia Control in Children with Urea Cycle Disorders (UCDs); Phase 2 Comparison of Sodium Phenylbutyrate and Glycerol Phenylbutyrate
Twenty four hour ammonia profiles and correlates of drug effect were examined in a phase…
CertaraAugust 1, 2011
Pharmacokinetics of Clofarabine in Patients with High-risk Inherited Metabolic Disorders Undergoing Brain-sparing Hematopoietic Cell Transplantation
Clofarabine, a newer purine analog with reduced central nervous system toxicity, may prove advantageous in…
CertaraMay 1, 2011
Pharmacology and Safety of Glycerol Phenylbutyrate in Healthy Adults and Adults with Cirrhosis
Phenylbutyric acid (PBA), which is approved for treatment of urea cycle disorders (UCDs) as sodium…
CertaraJune 1, 2010
Population Pharmacokinetics of Teduglutide Following Repeated Subcutaneous Administrations in Healthy Participants and in Patients with Short Bowel Syndrome and Crohn’s Disease
Teduglutide is a GLP-2 analog currently evaluated for the treatment of short bowel syndrome, Crohn's…
CertaraJanuary 1, 2010
Clinical Trial Simulations in Pediatric Patients Using Realistic Covariates: Application to Teduglutide, a Glucagon-like Peptide-2 Analog in Neonates and Infants with Short-bowel Syndrome
Teduglutide, a synthetic glucagon-like peptide-2 (GLP-2) analog with activity relating to the regeneration, maintenance, and…
CertaraDecember 1, 2009
