| Title |
Glycogen storage disease types I and II: Treatment updates
|
|---|---|
| Published in |
Journal of Inherited Metabolic Disease, February 2007
|
| DOI | 10.1007/s10545-007-0519-9 |
| Pubmed ID | |
| Authors |
D. D. Koeberl, P. S. Kishnani, Y. T. Chen |
| Abstract |
Prior to 2006 therapy for glycogen storage diseases consisted primarily of dietary interventions, which in the case of glycogen storage disease (GSD) type II (GSD II; Pompe disease) remained essentially palliative. Despite improved survival and growth, long-term complications of GSD type I (GSD I) have not responded to dietary therapy with uncooked cornstarch or continuous gastric feeding. The recognized significant risk of renal disease and liver malignancy in GSD I has prompted efforts towards curative therapy, including organ transplantation, in those deemed at risk. Results of clinical trials in infantile Pompe disease with alglucosidase alfa (Myozyme) showed prolonged survival reversal of cardiomyopathy, and motor gains. This resulted in broad label approval of Myozyme for Pompe disease in 2006. Furthermore, the development of experimental therapies, such as adeno-associated virus (AAV) vector-mediated gene therapy, holds promise for the availability of curative therapy in GSD I and GSD II/Pompe disease in the future. |
Mendeley readers
The data shown below were compiled from readership statistics for 72 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 3 | 4% |
| Spain | 1 | 1% |
| Unknown | 68 | 94% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Researcher | 12 | 17% |
| Student > Master | 11 | 15% |
| Student > Ph. D. Student | 9 | 13% |
| Student > Bachelor | 6 | 8% |
| Professor > Associate Professor | 3 | 4% |
| Other | 9 | 13% |
| Unknown | 22 | 31% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 18 | 25% |
| Agricultural and Biological Sciences | 11 | 15% |
| Biochemistry, Genetics and Molecular Biology | 9 | 13% |
| Nursing and Health Professions | 3 | 4% |
| Pharmacology, Toxicology and Pharmaceutical Science | 1 | 1% |
| Other | 7 | 10% |
| Unknown | 23 | 32% |
Attention Score in Context
This research output has an Altmetric Attention Score of 6. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 09 January 2024.
All research outputs
#5,372,093
of 25,177,382 outputs
Outputs from Journal of Inherited Metabolic Disease
#355
of 2,005 outputs
Outputs of similar age
#17,136
of 89,162 outputs
Outputs of similar age from Journal of Inherited Metabolic Disease
#3
of 12 outputs
Altmetric has tracked 25,177,382 research outputs across all sources so far. Compared to these this one has done well and is in the 75th percentile: it's in the top 25% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 2,005 research outputs from this source. They receive a mean Attention Score of 4.8. This one has done well, scoring higher than 77% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 89,162 tracked outputs that were published within six weeks on either side of this one in any source. This one has gotten more attention than average, scoring higher than 70% of its contemporaries.
We're also able to compare this research output to 12 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 75% of its contemporaries.