Sign Up to like & get
recommendations!
2
Published in 2022 at "Journal of Paediatrics and Child Health"
DOI: 10.1111/jpc.16165
Abstract: This is the first of three articles exploring the aspects of clinical care for children with rare neurological disorders including uncertainties old and new. The disruptive technologies of genomic sequencing and advanced therapeutics such as…
read more here.
Keywords:
children rare;
parents children;
neurological disorders;
rare neurological ... See more keywords
Sign Up to like & get
recommendations!
1
Published in 2021 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-020-01641-z
Abstract: Background Patient involvement in research increases the impact of research and the likelihood of adoption in clinical practice. A first step is to know which research themes are important for patients. We distributed a survey…
read more here.
Keywords:
patient representatives;
research;
research priorities;
disease ... See more keywords
Sign Up to like & get
recommendations!
1
Published in 2022 at "Frontiers in Pediatrics"
DOI: 10.3389/fped.2022.963601
Abstract: Objective To clarify the current state of methodology of clinical trials for rare neurological diseases in children, and to provide a basis for the further optimization of the trial design. Methods Data of clinical trials…
read more here.
Keywords:
clinical trials;
methodology;
trials rare;
rare neurological ... See more keywords
Sign Up to like & get
recommendations!
1
Published in 2022 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms23073946
Abstract: Rare diseases are those which affect a small number of people compared to the general population. However, many patients with a rare disease remain undiagnosed, and a large majority of rare diseases still have no…
read more here.
Keywords:
modeling rare;
neurological disorders;
rare neurological;
disease modeling ... See more keywords