Q. Dinh

Safety and efficacy of lenabasum in a phase 2 randomized, placebo-controlled trial in adults with cystic fibrosis.

J. Elborn, C. Colombo, P. Flume, 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

Treatment-naïve Gaucher disease patients achieve therapeutic goals and normalization with velaglucerase alfa by 4years in phase 3 trials.

D. Elstein, A. Zimran, D. Gonzalez, 2018, Blood cells, molecules & diseases.

Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trials.

S. Eichler, Y. Qiu, D. Elstein, 2017, Molecular genetics and metabolism.

A randomised, double-blind, placebo-controlled phase 3 study of lenabasum in diffuse cutaneous systemic sclerosis: RESOLVE-1 design and rationale.

M. Baron, D. Furst, C. Denton, 2021, Clinical and experimental rheumatology.

GS-14-ENVISION, a phase 3 study to evaluate efficacy and safety of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1, in acute hepatic porphyria patients

H. Bonkovsky, P. Stein, L. Gouya, 2019, Journal of Hepatology.

Inebilizumab for treatment of neuromyelitis optica spectrum disorder.

S. Vuocolo, A. Zabeti, Q. Dinh, 2021, Neurodegenerative disease management.

Development of anti-velaglucerase alfa antibodies in clinical trial-treated patients with Gaucher disease.

H. Ida, Y. Qiu, G. Pastores, 2016, Blood cells, molecules & diseases.

Efficacy and Safety of Lenabasum, a Cannabinoid Type 2 Receptor Agonist, in a Phase 3 Randomized Trial in Diffuse Cutaneous Systemic Sclerosis

M. Mayes, J. D. de Vries-Bouwstra, E. Lee, 2023, Arthritis & rheumatology.

FRI-442-Acute hepatic porphyria disease manifestations and daily life impacts in EXPLORE international, prospective, natural history study

H. Bonkovsky, P. Stein, S. Sandberg, 2019, Journal of Hepatology.