Rivista di formazione e aggiornamento professionale del pediatra e del medico di base, fondata nel 1982. In collaborazione con l'Associazione Culturale Pediatri.
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new therapies

2 articoli — 2006-2012

RI Aggiornamento
Nuovi concetti nel campo della distrofia muscolare di Duchenne

M. Pane, F. Bianco, C. Palermo, A. Graziano, E. Mercuri

2012/7 — pag. 429-435

Duchenne muscular dystrophy has an incidence of 1 in 3,500 male live births and is the most common muscular dystrophy in childhood. The “typical” course of the disease is characterized by progression of muscle weakness and contractures leading to l...

RI Focus
Prospettive di nuove terapie della fibrosi cistica

M. Conese, L. Palmieri, E. Copreni

2006/3 — pag. 155-173

Although to date symptomatic therapies for cystic fibrosis (CF) - based on the eradication/ control of opportunistic infections and facilitation of mucus excretion - have rapidly and significantly increased patients survival, an aetiological therapy ...

Argomenti correlati
Cystic Fibrosis (132) Respiratory Insufficiency (38) Genetic Therapy (27) Muscular Dystrophy, Duchenne (21) gene therapy (20) Cystic Fibrosis Transmembrane Conductance Regulator (18) Duchenne muscular dystrophy (18) terapia genica (17) Distrofia muscolare di Duchenne (13) research (10) Bone marrow (10) Pharmacogenetics (10) Duchenne Muscular Dystrophy (DMD) (8) Cardiomyopathies (8) Stem Cell Transplantation (7) ricerca (7) Terapie innovative (5) distrofina (4) Survival (4) Dystrophin (4)