Last Updated: 09/26/2026
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Found 19 publications
Advancing drug development for the prevention of spontaneous preterm birth.
Journal: Journal of neonatal-perinatal medicine
Published: January 12, 2026
Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease.
Journal: Journal of inherited metabolic disease
Published: February 05, 2024
Evaluation of FDA Labeling Changes Related to PREA Safety-Waivers.
Journal: Therapeutic innovation & regulatory science
Published: March 15, 2023
Model-Informed Approach Supporting Approval of Nexviazyme (Avalglucosidase Alfa-ngpt) in Pediatric Patients with Late-Onset Pompe Disease.
Journal: The AAPS journal
Published: September 22, 2022
Regulatory news: Avalglucosidase alfa-ngpt (Nexviazyme) for late-onset Pompe disease-FDA approval summary.
Journal: Journal of inherited metabolic disease
Published: August 05, 2022
Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases.
Journal: Orphanet journal of rare diseases
Published: October 18, 2020
Impact of Sex on Clinical Response in Rheumatoid Arthritis Patients Treated With Biologics at Approved Dosing Regimens.
Journal: Journal of clinical pharmacology
Published: May 14, 2020
FDA orphan products clinical trial grants: assessment of outcomes and impact on rare disease product development.
Journal: Orphanet journal of rare diseases
Published: April 29, 2020
Noninferiority Trials to Evaluate Drug Effects in Rheumatoid Arthritis.
Journal: Arthritis & rheumatology (Hoboken, N.J.)
Published: June 03, 2019
Dosage Considerations for Canakinumab in Children With Periodic Fever Syndromes.
Journal: Clinical pharmacology and therapeutics
Published: March 01, 2018
Dual-energy computed tomography has limited sensitivity for non-tophaceous gout: a comparison study with tophaceous gout.
Journal: BMC musculoskeletal disorders
Published: December 27, 2015
Temporal relationship between uric acid concentration and risk of diabetes in a community-based study population.
Journal: American journal of epidemiology
Published: January 15, 2014
Last Updated: 09/26/2026