Glycogen storage disease type II natural history, complications and prognosis
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief:
Overview
If left untreated, [#]% of patients with [disease name] may progress to develop [manifestation 1], [manifestation 2], and [manifestation 3].
OR
Common complications of [disease name] include [complication 1], [complication 2], and [complication 3].
OR
Prognosis is generally excellent/good/poor, and the 1/5/10-year mortality/survival rate of patients with [disease name] is approximately [#]%.
Natural History, Complications, and Prognosis
Natural History
- The symptoms of infantile glycogen storage disease type 2 (GSD type 2) usually develop in the first two months of life and start with symptoms such as cardiomegaly, cardiomyopathy, hypotonia, muscle weakness, respiratory distress, feeding difficulties, and failure to thrive.[1]
- Infantile GSD type 2 is usually diagnosed at a median age of 4.7 months.
- The symptoms of (disease name) typically develop ___ years after exposure to ___.
- If left untreated, patients with glycogen storage disease type 2 progress to cardiac insufficiency resulting in death within first two years of life.
Complications
- Common complications of [disease name] include:
- [Complication 1]
- [Complication 2]
- [Complication 3]
Prognosis
- Prognosis is generally excellent/good/poor, and the 1/5/10-year mortality/survival rate of patients with [disease name] is approximately [#]%.
- Depending on the extent of the [tumor/disease progression/etc.] at the time of diagnosis, the prognosis may vary. However, the prognosis is generally regarded as poor/good/excellent.
- The presence of [characteristic of disease] is associated with a particularly [good/poor] prognosis among patients with [disease/malignancy].
- [Subtype of disease/malignancy] is associated with the most favorable prognosis.
- The prognosis varies with the [characteristic] of tumor; [subtype of disease/malignancy] have the most favorable prognosis.
References
- ↑ Kishnani PS, Hwu WL, Mandel H, Nicolino M, Yong F, Corzo D; et al. (2006). "A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease". J Pediatr. 148 (5): 671–676. doi:10.1016/j.jpeds.2005.11.033. PMID 16737883.