Detalles de la búsqueda
1.
Mpox Diagnosis, Behavioral Risk Modification, and Vaccination Uptake among Gay, Bisexual, and Other Men Who Have Sex with Men, United Kingdom, 2022.
Emerg Infect Dis
; 30(5): 916-925, 2024 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-38573160
2.
Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy.
Mol Genet Metab
; 133(1): 100-108, 2021 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-33775523
3.
Measuring What Matters to Individuals with Angelman Syndrome and Their Families: Development of a Patient-Centered Disease Concept Model.
Child Psychiatry Hum Dev
; 52(4): 654-668, 2021 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-32880036
4.
Long-term Visual Outcomes after Release from Protocol in Patients who Participated in the Inhibition of VEGF in Age-related Choroidal Neovascularisation (IVAN) Trial.
Ophthalmology
; 127(9): 1191-1200, 2020 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-32359843
5.
Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis.
J Inherit Metab Dis
; 41(6): 1225-1233, 2018 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-29725868
6.
Clinical Outcome Assessments: Use of Normative Data in a Pediatric Rare Disease.
Value Health
; 21(5): 508-514, 2018 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-29753346
7.
Service Evaluation of an Exercise on Referral Scheme for Adults with Existing Health Conditions in the United Kingdom.
Int J Behav Med
; 25(3): 304-311, 2018 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-29143251
8.
Physical therapy management of infants and children with hypophosphatasia.
Mol Genet Metab
; 119(1-2): 14-9, 2016 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-27386757
9.
Correction to: Service Evaluation of an Exercise on Referral Scheme for Adults with Existing Health Conditions in the United Kingdom.
Int J Behav Med
; 28(6): 840, 2021 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-33880714
10.
Does brief telephone support improve engagement with a web-based weight management intervention? Randomized controlled trial.
J Med Internet Res
; 16(3): e95, 2014 Mar 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-24681761
11.
Long-term Retinal Morphology and Functional Associations in Treated Neovascular Age-Related Macular Degeneration: Findings from the Inhibition of VEGF in Age-Related Choroidal Neovascularisation Trial.
Ophthalmol Retina
; 6(8): 664-675, 2022 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-35314388
12.
Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients.
Orphanet J Rare Dis
; 16(1): 500, 2021 12 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-34857033
13.
Use of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) in X-Linked Myotubular Myopathy: Content Validity and Psychometric Performance.
J Neuromuscul Dis
; 8(1): 63-77, 2021.
Artículo
en Inglés
| MEDLINE | ID: mdl-32925083
14.
Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis.
Mol Genet Metab Rep
; 23: 100586, 2020 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-32292699
15.
Reliability and Validity of the 6-Minute Walk Test in Hypophosphatasia.
JBMR Plus
; 3(6): e10131, 2019 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-31346563
16.
The OMACS-PIL study: a randomised controlled trial within the OMACS observational study.
Trials
; 20(1): 772, 2019 Dec 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-31881927
17.
Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial.
Lancet Diabetes Endocrinol
; 7(2): 93-105, 2019 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-30558909
18.
Gratification, masturbation or paroxysmal hyperkinetic motor syndrome of infancy?
Can J Neurol Sci
; 40(3): 278-9, 2013 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-23603159
19.
Development and validation of a modified performance-oriented mobility assessment tool for assessing mobility in children with hypophosphatasia.
J Pediatr Rehabil Med
; 11(3): 187-192, 2018.
Artículo
en Inglés
| MEDLINE | ID: mdl-30223404
20.
Asfotase alfa therapy for children with hypophosphatasia.
JCI Insight
; 1(9): e85971, 2016 06 16.
Artículo
en Inglés
| MEDLINE | ID: mdl-27699270