SCOPUS 정보 검색 플랫폼

Volumn 9, Issue 3, 2017, Pages 289-292

Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis

(2) Monaco, Lucia a Faccio, Lucia a

a FONDAZIONE TELETHON (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

STRIMVELIS;

CELL THERAPY; CLINICAL RESEARCH; DRUG APPROVAL; DRUG MARKETING; GENE THERAPY; GENETIC DISORDER; GOOD CLINICAL PRACTICE; HUMAN; MEDICAL RESEARCH; MEDICATION COMPLIANCE; NOTE; RARE DISEASE; TRANSLATIONAL RESEARCH;

HUMANS; RARE DISEASES;

EID: 85011591313 PISSN: 17574676 EISSN: 17574684 Source Type: Journal
DOI: 10.15252/emmm.201607293 Document Type: Note

Times cited : (16)

References (8)

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2
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- Fighting rare diseases: the model of the telethon research institutes in Italy
- Ballabio A, Naldini L (2015) Fighting rare diseases: the model of the telethon research institutes in Italy. Hum Gene Ther 26: 183–185
- (2015) Hum Gene Ther , vol.26 , pp. 183-185
- Ballabio, A.¹ Naldini, L.²

3
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- LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
- Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E et al (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415–419
- (2003) Science , vol.302 , pp. 415-419
- Hacein-Bey-Abina, S.¹ Von Kalle, C.² Schmidt, M.³ McCormack, M.P.⁴ Wulffraat, N.⁵ Leboulch, P.⁶ Lim, A.⁷ Osborne, C.S.⁸ Pawliuk, R.⁹ Morillon, E.¹⁰

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- Reviewing in science requires quality criteria and professional reviewers
- Jurkat-Rott K, Lehmann-Horn F (2004) Reviewing in science requires quality criteria and professional reviewers. Eur J Cell Biol 83: 93–95
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- Jurkat-Rott, K.¹ Lehmann-Horn, F.²

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- (2016) Nature , vol.539 , pp. 150
- Naik, G.¹

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* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.