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Pediatric Neurology
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February 15, 2023
The Spectrum of MORC2-Related Disorders: A Potential Link to Cockayne Syndrome
Seth A Stafki, Johnnie Turner, Hannah R Littel, et al.
Journal of Neurology
|
May 5, 2016
Validation of a clinical practice-based algorithm for the diagnosis of autosomal recessive cerebellar ataxias based on NGS identified cases
Martial Mallaret, Mathilde Renaud, Claire Redin, et al.
American Journal of Human Genetics
|
August 22, 2017
Homozygous Truncating Variants in TBC1D23 Cause Pontocerebellar Hypoplasia and Alter Cortical Development
Ekaterina L Ivanova, Frédéric Tran Mau-Them, Saima Riazuddin, et al.
EMBO Molecular Medicine
|
June 6, 2020
Proteasome subunit PSMC3 variants cause neurosensory syndrome combining deafness and cataract due to proteotoxic stress
Ariane Kröll-Hermi, Frédéric Ebstein, Corinne Stoetzel, et al.
Orphanet Journal of Rare Diseases
|
March 24, 2016
Uncommon nucleotide excision repair phenotypes revealed by targeted high-throughput sequencing
Nadège Calmels, Géraldine Greff, Cathy Obringer, et al.
The Lancet. Neurology
|
September 22, 2023
Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Bettina C Henzi, Simone Schmidt, Sara Nagy, et al.
Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie
|
December 22, 2023
Effect of nusinersen after 3 years of treatment in 57 young children with SMA in terms of SMN2 copy number or type
Frédérique Audic, Sonia M Dubois, Julien Durigneux, et al.
Acta Neuropathologica Communications
|
July 9, 2022
Severe ACTA1-related nemaline myopathy: intranuclear rods, cytoplasmic bodies, and enlarged perinuclear space as characteristic pathological features on muscle biopsies
Clémence Labasse, Guy Brochier, Ana-Lia Taratuto, et al.
Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie
|
January 28, 2025
Evaluation of professional practices in the use of mexiletine for the management of childhood myotonia in French pediatric neuromuscular centers (MEXI-PEDI survey)
Sarah Barrière, Véronique Manel, Christine Barnerias, et al.
Orphanet Journal of Rare Diseases
|
June 14, 2020
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
Frédérique Audic, Marta Gomez Garcia de la Banda, Delphine Bernoux, et al.
Page
of 12
Search research articles
Search
Showing results (81-90 of 111) with videos related to
Sort By:
Page
of 12
Pediatric Neurology
|
February 15, 2023
The Spectrum of MORC2-Related Disorders: A Potential Link to Cockayne Syndrome
Seth A Stafki, Johnnie Turner, Hannah R Littel, et al.
Journal of Neurology
|
May 5, 2016
Validation of a clinical practice-based algorithm for the diagnosis of autosomal recessive cerebellar ataxias based on NGS identified cases
Martial Mallaret, Mathilde Renaud, Claire Redin, et al.
American Journal of Human Genetics
|
August 22, 2017
Homozygous Truncating Variants in TBC1D23 Cause Pontocerebellar Hypoplasia and Alter Cortical Development
Ekaterina L Ivanova, Frédéric Tran Mau-Them, Saima Riazuddin, et al.
EMBO Molecular Medicine
|
June 6, 2020
Proteasome subunit PSMC3 variants cause neurosensory syndrome combining deafness and cataract due to proteotoxic stress
Ariane Kröll-Hermi, Frédéric Ebstein, Corinne Stoetzel, et al.
Orphanet Journal of Rare Diseases
|
March 24, 2016
Uncommon nucleotide excision repair phenotypes revealed by targeted high-throughput sequencing
Nadège Calmels, Géraldine Greff, Cathy Obringer, et al.
The Lancet. Neurology
|
September 22, 2023
Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
Bettina C Henzi, Simone Schmidt, Sara Nagy, et al.
Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie
|
December 22, 2023
Effect of nusinersen after 3 years of treatment in 57 young children with SMA in terms of SMN2 copy number or type
Frédérique Audic, Sonia M Dubois, Julien Durigneux, et al.
Acta Neuropathologica Communications
|
July 9, 2022
Severe ACTA1-related nemaline myopathy: intranuclear rods, cytoplasmic bodies, and enlarged perinuclear space as characteristic pathological features on muscle biopsies
Clémence Labasse, Guy Brochier, Ana-Lia Taratuto, et al.
Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie
|
January 28, 2025
Evaluation of professional practices in the use of mexiletine for the management of childhood myotonia in French pediatric neuromuscular centers (MEXI-PEDI survey)
Sarah Barrière, Véronique Manel, Christine Barnerias, et al.
Orphanet Journal of Rare Diseases
|
June 14, 2020
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
Frédérique Audic, Marta Gomez Garcia de la Banda, Delphine Bernoux, et al.
Page
of 12