1 |
In vitro correction of ARSA deficiency in human skin fibroblasts from Metachromatic Leukodystrophy patients after treatment with microencapsulated recombinant cells |
|
|
| Lagranha, V.L., Baldo, G., De Carvalho, T.G., Burin, M., Saraiva-Pereira, M.L., Matte, U., Giugliani, R. |
|
| Metabolic Brain Disease. 2008; 23(4): 469-484 |
|
| [Pubmed] [Google Scholar] |
|
2 |
Therapy of adult Gaucher disease |
|
|
| Schmitz J, Poll LW, vom Dahl S |
|
| HAEMATOLOGICA-THE HEMATOLOGY JOURNAL. 2007; 92 (2): 148-152 |
|
| [Pubmed] [Google Scholar] |
|
3 |
Low frequency maintenance therapy with imiglucerase in adult type I Gaucher disease: a prospective randomized controlled trial |
|
|
| de Fost M, Aerts JMFG, Groener JEM, et al. |
|
| HAEMATOLOGICA-THE HEMATOLOGY JOURNAL. 2007; 92 (2): 215-221 |
|
| [Pubmed] [Google Scholar] |
|
4 |
Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts |
|
|
| Consiglio A, Martino S, Dolcetta D, et al. |
|
| JOURNAL OF THE NEUROLOGICAL SCIENCES. 2007; 255 (1-2): 7-16 |
|
| [Pubmed] [Google Scholar] |
|
5 |
Short-term effect of miglustat in every day clinical use in treatment-naive or previously treated patients with type 1 Gauchers disease |
|
|
| Giraldo P, Latre P, Alfonso P, et al. |
|
| HAEMATOLOGICA-THE HEMATOLOGY JOURNAL. 2006; 91 (5): 703-706 |
|
| [Pubmed] [Google Scholar] |
|
6 |
Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts |
|
|
| Friso A, Tomanin R, Alba S, et al. |
|
| JOURNAL OF GENE MEDICINE. 2005; 7 (11): 1482-1491 |
|
| [Pubmed] [Google Scholar] |
|
7 |
Fabry disease: diagnosis and management, with emphasis on the renal manifestations |
|
|
| Warnock DG |
|
| CURRENT OPINION IN NEPHROLOGY AND HYPERTENSION. 2005; 14 (2): 87-95 |
|
| [Pubmed] [Google Scholar] |
|
8 |
The need to define guidelines for maintenance enzyme replacement therapy for Gaucher disease: Expanded intervals, dose modification, or drug vacations? |
|
|
| Zimran, A., Elstein, D. |
|
| Journal of Postgraduate Medicine. 2003; 49(2): 130-131 |
|
| [Pubmed] [Google Scholar] |
|