Protocol for initiation of miglustat treatment in NP-C. Reproduced
Por um escritor misterioso
Descrição
Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study, Orphanet Journal of Rare Diseases
Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study, Orphanet Journal of Rare Diseases
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Miglustat
F-1/A
Protocol for initiation of miglustat treatment in NP-C. Reproduced with
Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases
Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice: Molecular Therapy
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por adulto (o preço varia de acordo com o tamanho do grupo)
