Publication & Citation Trends
Publications
240 total
Vosoritide (BMN 111) in Children with Achondroplasia: Initial results from a Phase 2, open-label, sequential cohort, dose-escalation study
Cited by 0
Semantic Scholar
Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease PDF
Cited by 358
OpenAlex
Approach to management of cross-reactive immunologic material (CRIM)-negative infantile pompe patients treated with ERT: Role of immune modulation in changing the natural history
Cited by 1
Semantic Scholar
Sustained, Long-Term Renal Stabilization After 54 Months of Agalsidase β Therapy in Patients with Fabry Disease
Cited by 435
OpenAlex
Fabry disease: Baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry
Cited by 384
OpenAlex
A rational approach for a phase I clinical study design to evaluate CNTO 95, a novel, fully human anti-avmonoclonal antibody (MAb), in patients with solid tumors.
Cited by 3
Semantic Scholar
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry
Cited by 541
OpenAlex
The Gaucher Registry
Cited by 583
OpenAlex
Research Topics
Lysosomal Storage Disorders Research
(92)
Neurofibromatosis and Schwannoma Cases
(35)
Carbohydrate Chemistry and Synthesis
(26)
Connective tissue disorders research
(26)
Glycosylation and Glycoproteins Research
(24)
Affiliations
Northwestern University
Children's Hospital of Wisconsin
University of Maryland, Baltimore
Lurie Children's Hospital
Royal Children's Hospital