Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis.
about
Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem.Forkhead-Box-P3 Gene Transfer in Human CD4+ T Conventional Cells for the Generation of Stable and Efficient Regulatory T Cells, Suitable for Immune Modulatory Therapy.Impact of biobanks on research outcomes in rare diseases: a systematic review
P2860
Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis.
description
2017 nî lūn-bûn
@nan
2017年の論文
@ja
2017年論文
@yue
2017年論文
@zh-hant
2017年論文
@zh-hk
2017年論文
@zh-mo
2017年論文
@zh-tw
2017年论文
@wuu
2017年论文
@zh
2017年论文
@zh-cn
name
Patient-driven search for rare ...... trategy leading to Strimvelis.
@en
Patient-driven search for rare ...... trategy leading to Strimvelis.
@nl
type
label
Patient-driven search for rare ...... trategy leading to Strimvelis.
@en
Patient-driven search for rare ...... trategy leading to Strimvelis.
@nl
prefLabel
Patient-driven search for rare ...... trategy leading to Strimvelis.
@en
Patient-driven search for rare ...... trategy leading to Strimvelis.
@nl
P2860
P356
P1476
Patient-driven search for rare ...... trategy leading to Strimvelis.
@en
P2093
Lucia Faccio
P2860
P304
P356
10.15252/EMMM.201607293
P50
P577
2017-02-01T00:00:00Z