Browse by UCL people
Group by: Type | Date
Jump to: Article
Number of items: 4.
Article
Chesshyre, Mary;
Ridout, Deborah;
Hashimoto, Yasumasa;
Ookubo, Yoko;
Torelli, Silvia;
Maresh, Kate;
Ricotti, Valeria;
... Muntoni, Francesco; + view all
(2022)
Investigating the role of dystrophin isoform deficiency in motor function in Duchenne muscular dystrophy.
Journal of Cachexia, Sarcopenia and Muscle
10.1002/jcsm.12914.
|
 |
|
Coratti, G;
Pane, M;
Brogna, C;
Ricotti, V;
Messina, S;
D'Amico, A;
Bruno, C;
... on behalf on the International DMD Group and the iMDEX Consortiu; + view all
(2021)
North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up.
PLoS One
, 16
(6)
, Article e0253882. 10.1371/journal.pone.0253882.
|
|
|
Mercuri, E;
Seferian, AM;
Servais, L;
Deconinck, N;
Stevenson, H;
Ni, X;
Zhang, W;
... Muntoni, F; + view all
(2023)
Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping.
Neuromuscular Disorders
, 33
(6)
pp. 476-483.
10.1016/j.nmd.2023.03.008.
|
 |
|
Stimpson, G;
Chesshyre, M;
Baranello, G;
Muntoni, F;
(2021)
Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation.
Frontiers in Genetics
, 12
, Article 759994. 10.3389/fgene.2021.759994.
|
|
