Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics

Richard S. Finkel, Eugenio Mercuri, Oscar H. Meyer, Anita K. Simonds, Mary K. Schroth, Robert J. Graham, Janbernd Kirschner, Susan T. Iannaccone, Thomas O. Crawford, Simon Woods, Francesco Muntoni, Brunhilde Wirth, Jacqueline Montes, Marion Main, Elena S. Mazzone, Michael Vitale, Brian Snyder, Susana Quijano-Roy, Enrico Bertini, Rebecca Hurst DavisYing Qian, Thomas Sejersen

Research output: Contribution to journalArticle

55 Citations (Scopus)

Abstract

This is the second half of a two-part document updating the standard of care recommendations for spinal muscular atrophy published in 2007. This part includes updated recommendations on pulmonary management and acute care issues, and topics that have emerged in the last few years such as other organ involvement in the severe forms of spinal muscular atrophy and the role of medications. Ethical issues and the choice of palliative versus supportive care are also addressed. These recommendations are becoming increasingly relevant given recent clinical trials and the prospect that commercially available therapies will likely change the survival and natural history of this disease.

Original languageEnglish (US)
JournalNeuromuscular Disorders
DOIs
StateAccepted/In press - Jan 1 2018

Fingerprint

Spinal Muscular Atrophy
Ethics
Immunization
Lung
Standard of Care
Clinical Trials
Therapeutics

Keywords

  • Acute
  • Care
  • Nutrition
  • Pulmonary
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)

Cite this

Diagnosis and management of spinal muscular atrophy : Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. / Finkel, Richard S.; Mercuri, Eugenio; Meyer, Oscar H.; Simonds, Anita K.; Schroth, Mary K.; Graham, Robert J.; Kirschner, Janbernd; Iannaccone, Susan T.; Crawford, Thomas O.; Woods, Simon; Muntoni, Francesco; Wirth, Brunhilde; Montes, Jacqueline; Main, Marion; Mazzone, Elena S.; Vitale, Michael; Snyder, Brian; Quijano-Roy, Susana; Bertini, Enrico; Davis, Rebecca Hurst; Qian, Ying; Sejersen, Thomas.

In: Neuromuscular Disorders, 01.01.2018.

Research output: Contribution to journalArticle

Finkel, RS, Mercuri, E, Meyer, OH, Simonds, AK, Schroth, MK, Graham, RJ, Kirschner, J, Iannaccone, ST, Crawford, TO, Woods, S, Muntoni, F, Wirth, B, Montes, J, Main, M, Mazzone, ES, Vitale, M, Snyder, B, Quijano-Roy, S, Bertini, E, Davis, RH, Qian, Y & Sejersen, T 2018, 'Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics', Neuromuscular Disorders. https://doi.org/10.1016/j.nmd.2017.11.004
Finkel, Richard S. ; Mercuri, Eugenio ; Meyer, Oscar H. ; Simonds, Anita K. ; Schroth, Mary K. ; Graham, Robert J. ; Kirschner, Janbernd ; Iannaccone, Susan T. ; Crawford, Thomas O. ; Woods, Simon ; Muntoni, Francesco ; Wirth, Brunhilde ; Montes, Jacqueline ; Main, Marion ; Mazzone, Elena S. ; Vitale, Michael ; Snyder, Brian ; Quijano-Roy, Susana ; Bertini, Enrico ; Davis, Rebecca Hurst ; Qian, Ying ; Sejersen, Thomas. / Diagnosis and management of spinal muscular atrophy : Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. In: Neuromuscular Disorders. 2018.
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