Results 311 to 320 of about 121,613 (384)

Identification and Phylogenetic Characterisation of Novel Adeno‐Associated Virus Capsids in Non‐Human Primate Tissues

Cell Proliferation, EarlyView.
AAV's widespread application is currently confronting critical challenges. This study established a systematic framework of ‘natural discovery–evolutionary analysis–functional optimization’, providing a new paradigm for the development of next‐generation AAV vectors with clinical‐grade tissue specificity, low immunogenicity, and cross‐species ...
Liyu Zhu, Kai Xu, Yali Ding, Kailun Liu, Jing Liu, Zongren Hou, Rui Niu, Ning Yang, Hualing Qin, Baoyang Hu, Ying Zhang, Wei Li   +11 more
wiley   +1 more source

Effects of physical therapy on motor ability in patients with spinal muscular atrophy III: a study protocol for a randomised controlled trial. [PDF]

BMJ Open
Zhang J, Wang B, He J, Zhu L, Dong X.
europepmc   +1 more source

Expressive language and social communication abilities in children with spinal muscular atrophy type 1

Developmental Medicine &Child Neurology, EarlyView.
Abstract Aim To investigate parent‐reported expressive language and social communication abilities in children with spinal muscular atrophy type 1 (SMA1) treated with disease‐modifying therapies. Method This was a cross‐sectional feasibility study performed at the Dubowitz Neuromuscular Centre, London (UK), and the Centro Clinico Nemo Pediatrico, Rome (
Chiara Brusa, Bianca Buchignani, Chiara Cutri, Giorgia Coratti, Elaine Clark, Emily Johnson, Nikki Cornell, Mariacristina Scoto, Marika Pane, Eugenio Maria Mercuri, Francesco Muntoni, Giovanni Baranello, The Language and Social Communication in SMA working group, Harriet Weststrate, Emily Barritt, Laura Antonaci, Daniela Leone, Concetta Palermo, Pinki Munot, Adnan Manzur   +19 more
wiley   +1 more source

Drug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO-10-27. [PDF]

J Cachexia Sarcopenia Muscle
Bézier C, Cottin S, Hashemi PN, El Khoury M, Clerc Z, Balducci C, Sapaly D, Weill L, Lafont R, Veillet S, Dilda PJ, Charbonnier F, Latil M, Biondi O.   +13 more
europepmc   +1 more source

Participation, environment, and quality of life in children with cerebral palsy and physical disabilities

Developmental Medicine &Child Neurology, EarlyView.
Abstract Aim To compare and examine relationships between participation, environmental factors, and quality of life (QoL) in children with cerebral palsy and physical disability (CP/PD) and typically developing children. Method Participants were 59 children (6–12 years; 31 females): 30 with CP/PD (mean age 8 years 7 months, standard deviation 1 year 6 ...
Nava Gelkop, Batya Engel‐Yeger
wiley   +1 more source

Proband-independent noninvasive prenatal diagnosis for spinal muscular atrophy: early detection paving the way for early prenatal treatment. [PDF]

Ann Med
Li H, Gao S, Li S, Zhao Z, Fu X, Zhu J, Feng J, Tang W, Wu D, Kong X.   +9 more
europepmc   +1 more source

The Children's Eating and Drinking Activity Scale: Interrater reliability, concurrent validity, and responsiveness

Developmental Medicine &Child Neurology, EarlyView.
This paper evaluated the interrater reliability, concurrent validity and responsiveness of the Children's Eating and Drinking Activity Scale ‐ a measure of functional outcome for children with paediatric feeding disorder. Abstract Aim To assess the interrater reliability, concurrent validity, and responsiveness of the Children's Eating and Drinking ...
Ericka Joinelle Mantaring, Sheung Yu Sylfiana Wong, Michelle McInerney, Vicky Thorpe, Alexandra Stewart   +4 more
wiley   +1 more source

Longitudinal efficacy of risdiplam treatment in Chinese children with spinal muscular atrophy. [PDF]

Orphanet J Rare Dis
Yan Y, Zhu D, Ma K, Luo X, Zhang X, Deng X, Lu M, Li Y, Li J, Gao F, Mao S.   +10 more
europepmc   +1 more source

Estimation Of The Quality Of Life Benefits Associated With Treatment For Spinal Muscular Atrophy

, 2019
Lloyd AJ, Robin Thompson, Katy Gallop, Megan Teynor   +3 more
openalex   +1 more source