Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial.
Roberto GiuglianiLuciana GiuglianiFabiano de Oliveira PoswarKarina Carvalho DonisAmauri Dalla CorteMathias SchmidtRuben J BoadoIgor NestrasilCarol NguyenSteven ChenWilliam M PardridgePublished in: Orphanet journal of rare diseases (2018)
Clinical Trials.Gov, NCT03053089 . Retrospectively registered 9 February, 2017; Clinical Trials.Gov, NCT03071341 . Registered 6 March, 2017.
Keyphrases
- clinical trial
- type diabetes
- phase ii
- resting state
- white matter
- early onset
- open label
- double blind
- bipolar disorder
- functional connectivity
- glycemic control
- randomized controlled trial
- study protocol
- copy number
- gestational age
- multiple sclerosis
- gene expression
- weight loss
- blood brain barrier
- skeletal muscle
- preterm birth
- smoking cessation