Medical management of muscle weakness in Duchenne muscular dystrophy.
<h4>Introduction</h4>Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens.<h4>Methods</h...
| Main Authors: | , , , , , , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Public Library of Science (PLoS)
2020-01-01
|
| Series: | PLoS ONE |
| Online Access: | https://doi.org/10.1371/journal.pone.0240687 |
| _version_ | 1818740724727808000 |
|---|---|
| author | Sarah R Rivera Sumit K Jhamb Hoda Z Abdel-Hamid Gyula Acsadi John Brandsema Emma Ciafaloni Basil T Darras Susan T Iannaccone Chamindra G Konersman Nancy L Kuntz Craig M McDonald Julie A Parsons Carolina Tesi Rocha Craig M Zaidman Russell J Butterfield Anne M Connolly Katherine D Mathews |
| author_facet | Sarah R Rivera Sumit K Jhamb Hoda Z Abdel-Hamid Gyula Acsadi John Brandsema Emma Ciafaloni Basil T Darras Susan T Iannaccone Chamindra G Konersman Nancy L Kuntz Craig M McDonald Julie A Parsons Carolina Tesi Rocha Craig M Zaidman Russell J Butterfield Anne M Connolly Katherine D Mathews |
| author_sort | Sarah R Rivera |
| collection | DOAJ |
| description | <h4>Introduction</h4>Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens.<h4>Methods</h4>A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists' responses were collected using a modified Delphi approach.<h4>Results</h4>Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid.<h4>Discussion</h4>The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects. |
| first_indexed | 2024-12-18T01:45:17Z |
| format | Article |
| id | doaj.art-6715df668b4d4feb89a01ef4461b2d2e |
| institution | Directory Open Access Journal |
| issn | 1932-6203 |
| language | English |
| last_indexed | 2024-12-18T01:45:17Z |
| publishDate | 2020-01-01 |
| publisher | Public Library of Science (PLoS) |
| record_format | Article |
| series | PLoS ONE |
| spelling | doaj.art-6715df668b4d4feb89a01ef4461b2d2e2022-12-21T21:25:12ZengPublic Library of Science (PLoS)PLoS ONE1932-62032020-01-011510e024068710.1371/journal.pone.0240687Medical management of muscle weakness in Duchenne muscular dystrophy.Sarah R RiveraSumit K JhambHoda Z Abdel-HamidGyula AcsadiJohn BrandsemaEmma CiafaloniBasil T DarrasSusan T IannacconeChamindra G KonersmanNancy L KuntzCraig M McDonaldJulie A ParsonsCarolina Tesi RochaCraig M ZaidmanRussell J ButterfieldAnne M ConnollyKatherine D Mathews<h4>Introduction</h4>Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens.<h4>Methods</h4>A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists' responses were collected using a modified Delphi approach.<h4>Results</h4>Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid.<h4>Discussion</h4>The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.https://doi.org/10.1371/journal.pone.0240687 |
| spellingShingle | Sarah R Rivera Sumit K Jhamb Hoda Z Abdel-Hamid Gyula Acsadi John Brandsema Emma Ciafaloni Basil T Darras Susan T Iannaccone Chamindra G Konersman Nancy L Kuntz Craig M McDonald Julie A Parsons Carolina Tesi Rocha Craig M Zaidman Russell J Butterfield Anne M Connolly Katherine D Mathews Medical management of muscle weakness in Duchenne muscular dystrophy. PLoS ONE |
| title | Medical management of muscle weakness in Duchenne muscular dystrophy. |
| title_full | Medical management of muscle weakness in Duchenne muscular dystrophy. |
| title_fullStr | Medical management of muscle weakness in Duchenne muscular dystrophy. |
| title_full_unstemmed | Medical management of muscle weakness in Duchenne muscular dystrophy. |
| title_short | Medical management of muscle weakness in Duchenne muscular dystrophy. |
| title_sort | medical management of muscle weakness in duchenne muscular dystrophy |
| url | https://doi.org/10.1371/journal.pone.0240687 |
| work_keys_str_mv | AT sarahrrivera medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT sumitkjhamb medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT hodazabdelhamid medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT gyulaacsadi medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT johnbrandsema medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT emmaciafaloni medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT basiltdarras medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT susantiannaccone medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT chamindragkonersman medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT nancylkuntz medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT craigmmcdonald medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT julieaparsons medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT carolinatesirocha medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT craigmzaidman medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT russelljbutterfield medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT annemconnolly medicalmanagementofmuscleweaknessinduchennemusculardystrophy AT katherinedmathews medicalmanagementofmuscleweaknessinduchennemusculardystrophy |