✦ LIBER ✦

Early administration of enzyme replacement therapy for Pompe disease: Short-term follow-up results

✍ Scribed by M. A. Hamdan; M. H. Almalik; H. M. Mirghani

Book ID: 106374433
Publisher: Springer
Year: 2008
Tongue: English
Weight: 217 KB
Volume: 31
Category: Article
ISSN: 0141-8955
DOI: 10.1007/s10545-008-1000-0

No coin nor oath required. For personal study only.

📜 SIMILAR VOLUMES

Cognitive and adaptive functioning of ch

Cognitive and adaptive functioning of children with infantile Pompe disease treated with enzyme replacement therapy: Long-term follow-up

✍ Gail A. Spiridigliozzi; James H. Heller; Priya S. Kishnani 📂 Article 📅 2012 🏛 John Wiley and Sons 🌐 English ⚖ 100 KB 👁 2 views

Immune tolerance induction to enzyme-rep

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model

✍ A. Joseph; K. Munroe; M. Housman; R. Garman; S. Richards 📂 Article 📅 2008 🏛 John Wiley and Sons 🌐 English ⚖ 303 KB

Long-term follow-up results of postopera

Long-term follow-up results of postoperative radiation therapy for Cushing’s disease

✍ Giuseppe Minniti; Mattia Osti; Marie Lise Jaffrain-Rea; Vincenzo Esposito; Giamp 📂 Article 📅 2007 🏛 Springer US 🌐 English ⚖ 205 KB

A novel mutation of the GAA gene in a Fi

A novel mutation of the GAA gene in a Finnish late-onset pompe disease patient: Clinical phenotype and follow-up with enzyme replacement therapy

✍ Mari P. Korpela; Anders Paetau; Mervi I. Löfberg; Marjut H. Timonen; Antti E. La 📂 Article 📅 2009 🏛 John Wiley and Sons 🌐 English ⚖ 139 KB

## Abstract Pompe disease is a rare, progressive disease leading to skeletal muscle weakness due to deficiency of the acid α‐glucosidase (GAA) enzyme. Herein we report the first diagnosed Finnish patient with a phenotype compatible with the late‐onset form of Pompe disease. Molecular genetic analys