| Title |
Double blind placebo control trial of large neutral amino acids in treatment of PKU: Effect on blood phenylalanine
|
|---|---|
| Published in |
Journal of Inherited Metabolic Disease, February 2007
|
| DOI | 10.1007/s10545-007-0556-4 |
| Pubmed ID | |
| Authors |
R. Matalon, K. Michals‐Matalon, G. Bhatia, A. B. Burlina, A. P. Burlina, C. Braga, L. Fiori, M. Giovannini, E. Grechanina, P. Novikov, J. Grady, S. K. Tyring, F. Guttler |
| Abstract |
Large neutral amino acids (LNAA) have been used on a limited number of patients with phenylketonuria (PKU) with the purpose of decreasing the influx of phenylalanine (Phe) to the brain. In an open-label study using LNAA, a surprising decline of blood Phe concentration was found in patients with PKU in metabolic treatment centres in Russia, the Ukraine, and the United States. To validate the data obtained from this trial, a short-term double-blind placebo control study was done using LNAA in patients with PKU, with the participation of three additional metabolic centres--Milan, Padua and Rio de Janeiro. The results of the short trial showed significant lowering of blood Phe concentration by an average of 39% from baseline. The data from the double-blind placebo control are encouraging, establishing proof of principle of the role of orally administered LNAA in lowering blood Phe concentrations in patients with PKU. Long-term studies will be needed to validate the acceptability, efficacy and safety of such treatment. |
Mendeley readers
The data shown below were compiled from readership statistics for 76 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Canada | 1 | 1% |
| Unknown | 75 | 99% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Bachelor | 13 | 17% |
| Researcher | 11 | 14% |
| Student > Postgraduate | 9 | 12% |
| Student > Master | 8 | 11% |
| Student > Ph. D. Student | 6 | 8% |
| Other | 11 | 14% |
| Unknown | 18 | 24% |
| Readers by discipline | Count | As % |
|---|---|---|
| Agricultural and Biological Sciences | 19 | 25% |
| Medicine and Dentistry | 17 | 22% |
| Biochemistry, Genetics and Molecular Biology | 7 | 9% |
| Nursing and Health Professions | 5 | 7% |
| Psychology | 2 | 3% |
| Other | 5 | 7% |
| Unknown | 21 | 28% |
Attention Score in Context
This research output has an Altmetric Attention Score of 3. 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 16 April 2019.
All research outputs
#7,451,284
of 22,780,165 outputs
Outputs from Journal of Inherited Metabolic Disease
#684
of 1,841 outputs
Outputs of similar age
#27,095
of 75,947 outputs
Outputs of similar age from Journal of Inherited Metabolic Disease
#3
of 10 outputs
Altmetric has tracked 22,780,165 research outputs across all sources so far. This one is in the 44th percentile – i.e., 44% of other outputs scored the same or lower than it.
So far Altmetric has tracked 1,841 research outputs from this source. They receive a mean Attention Score of 4.6. This one is in the 38th percentile – i.e., 38% of its peers scored the same or lower than it.
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