Future Rare Diseases

Research and Development of Oligonucleotide Therapeutics in Japan for Rare Diseases

Junetsu Igarashi, Yasuharu Niwa, Daisuke Sugiyama · 2022 · 27 citations · Full text

Pharmacy, Molecular Genetics, Pharmaceutical Biotechnology +9

The Global Role of Patients, Advocates and Caregivers in Rare Diseases

Mukund Nori, Dakota Fisher-Vance, Laura Wuerth et al. · 2022 · 16 citations · Full text

Nursing, Health Informatics, Advocacy +13

GALILEO-1: A Phase I/II Safety and Efficacy Study of FLT201 Gene Therapy for Gaucher Disease Type 1

Derralynn Hughes, Francesca Ferrante · 2023 · 14 citations · Full text

Life Sciences, Genetics, Molecular Biology +14

Nefecon (Targeted-Release Formulation-Budesonide) for the Treatment of IgA Nephropathy

Lucia Del Vecchio, Chiara Rimoldi, Claudio Pozzi · 2021 · 12 citations · Full text

Medicine, Nephrology, Pharmacology +15

Functional Genetics in Inborn Errors of Immunity

Emma Rey-Jurado, M. Cecilia Poli · 2021 · 11 citations · Full text

Life Sciences, Genetics, Immunology +16