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.

Finkel, R., Mercuri, E. M., Meyer, O., Simonds, A., Schroth, M., Graham, R., Kirschner, J., Iannaccone, S., Crawford, T., Woods, S., Muntoni, F., Wirth, B., Montes, J., Main, M., Mazzone, E. S., Vitale, M., Snyder, B., Quijano-Roy, S., Bertini, E. S., Davis, R., Qian, Y., Sejerse, N., Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics., <<NEUROMUSCULAR DISORDERS>>, 2018; (28 (3)): 197-207. [doi:10.1016/j.nmd.2017.11.004] [http://hdl.handle.net/10807/125804]

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

Mercuri, Eugenio Maria;Mazzone, Elena Stacy;Bertini, Enrico Silvio;
2018

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.
2018
Inglese
Finkel, R., Mercuri, E. M., Meyer, O., Simonds, A., Schroth, M., Graham, R., Kirschner, J., Iannaccone, S., Crawford, T., Woods, S., Muntoni, F., Wirth, B., Montes, J., Main, M., Mazzone, E. S., Vitale, M., Snyder, B., Quijano-Roy, S., Bertini, E. S., Davis, R., Qian, Y., Sejerse, N., Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics., <<NEUROMUSCULAR DISORDERS>>, 2018; (28 (3)): 197-207. [doi:10.1016/j.nmd.2017.11.004] [http://hdl.handle.net/10807/125804]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/125804
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