Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review
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M. Pineda | S. Kolb | P. Karimzadeh | V. Malinová | M. Kolníková | C. Velten | K. Juríčková | J. L. Insua | Stefan A. Kolb