This is a brochure created by the IPA and Genzyme to aid doctors in their understanding of Pompe Disease.

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
The download file is a printout of the study results web page at clinicaltrials.gov, dated from June 24, 2010.

Scientific publication

• van der Ploeg AT et al.
  A randomized study of alglucosidase alfa in late-onset Pompe's disease.
  N Engl J Med. 2010 Apr 15;362(15):1396-406

MG Mullender, NA Blom, M De Kleuver, JM Fock, WMGC Hitters, AMC Horemans, CJ Kalkman, JEH Pruijs, RR Timmer, PJ Titarsolej, NC Van Haasteren, MJ Van Tol-de Jager, AJ Van Vught and BJ Van Royen
Scoliosis 2008, 3:14 - Free Article (PDF, external link)

"In order to optimize the treatment for scoliosis in neuromuscular disorders a Dutch guideline has been composed. This evidence-based, multidisciplinary guideline addresses conservative treatment, the preoperative, perioperative, and postoperative care of scoliosis in neuromuscular disorders."

Johanna M.P. Van den Hout et al.
Pediatrics 113, No. 5, May 2004 - Free Article (PDF, external link)

"Our study shows that a safe and effective medicine can be produced in the milk of mammals and encourages additional development of enzyme replacement therapy for the several forms of Pompe disease. Restoration of skeletal muscle function and prevention of pulmonary insufficiency require dosing in the range of 20 to 40 mg/kg/week. The effect depends on residual muscle function at the start of treatment. Early start of treatment is required."

The article is available as a provisional PDF. The fully formatted PDF and HTML versions are in production.

Scientific publication

• Stephanie S Weinreich, Tessel Rigter, Carla G van El, Wybo J Dondorp, Pieter J Kostense, Ans T van der Ploeg, Arnold JJ Reuser, Martina C Cornel and Marloes LC Hagemans
  Public support for neonatal screening for Pompe disease, a broad-phenotype condition.
  Orphanet Journal of Rare Diseases 2012, 7:15
  doi:10.1186/1750-1172-7-15