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42 in total

1. Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.

Authors: Kai Qiu; Darin J Falk; Paul J Reier; Barry J Byrne; David D Fuller
Journal: Mol Ther Date: 2011-10-18 Impact factor: 11.454

Review 2. Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.

Authors: Marianne Rohrbach; Joe T R Clarke
Journal: Drugs Date: 2007 Impact factor: 9.546

Review 3. Muscle glycogenoses: an overview.

Authors: S Di Mauro
Journal: Acta Myol Date: 2007-07

4. Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease.

Authors: Cathryn S Mah; Darin J Falk; Sean A Germain; Jeffry S Kelley; Melissa A Lewis; Denise A Cloutier; Lara R DeRuisseau; Thomas J Conlon; Kerry O Cresawn; Thomas J Fraites; Martha Campbell-Thompson; David D Fuller; Barry J Byrne
Journal: Mol Ther Date: 2010-01-26 Impact factor: 11.454

Review 5. Pompe disease gene therapy.

Authors: Barry J Byrne; Darin J Falk; Christina A Pacak; Sushrusha Nayak; Roland W Herzog; Melissa E Elder; Shelley W Collins; Thomas J Conlon; Nathalie Clement; Brian D Cleaver; Denise A Cloutier; Stacy L Porvasnik; Saleem Islam; Mai K Elmallah; Anatole Martin; Barbara K Smith; David D Fuller; Lee Ann Lawson; Cathryn S Mah
Journal: Hum Mol Genet Date: 2011-04-25 Impact factor: 6.150

Review 6. An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond.

Authors: Aditi Korlimarla; Jeong-A Lim; Priya S Kishnani; Baodong Sun
Journal: Ann Transl Med Date: 2019-07

Nervous system in Pompe's disease. Ultrastructure and biochemistry.

1. Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice.

Review 2. Treatment of lysosomal storage disorders : progress with enzyme replacement therapy.

Review 3. Muscle glycogenoses: an overview.

4. Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease.

Review 5. Pompe disease gene therapy.

Review 6. An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond.

7. Pompe's disease: an inborn lysosomal disorder with storage of glycogen. A study of brain and striated muscle.

8. Rapidly Progressive White Matter Involvement in Early Childhood: The Expanding Phenotype of Infantile Onset Pompe?

9. Neuronal lipidosis and neuroaxonal dystrophy in cerebro-hepato-renal (Zellweger) syndrome.

10. A review of treatment of Pompe disease in infants.