TY - JOUR
T1 - Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics.
AU - Finkel, Richard S.
AU - Mercuri, Eugenio Maria
AU - Meyer, Oscar H.
AU - Simonds, Anita K.
AU - Schroth, Mary K.
AU - Graham, Robert J.
AU - Kirschner, Janbernd
AU - Iannaccone, Susan T.
AU - Crawford, Thomas O.
AU - Woods, Simon
AU - Muntoni, Francesco
AU - Wirth, Brunhilde
AU - Montes, Jacqueline
AU - Main, Marion
AU - Mazzone, Elena Stacy
AU - Vitale, Michael
AU - Snyder, Brian
AU - Quijano-Roy, Susana
AU - Bertini, Enrico
AU - Bertini, Enrico Silvio
AU - Davis, Rebecca Hurst
AU - Qian, Ying
AU - Sejersen, Thomas
PY - 2018
Y1 - 2018
N2 - 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.
AB - 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.
KW - spinal muscular atrophy
KW - spinal muscular atrophy
UR - http://hdl.handle.net/10807/125804
U2 - 10.1016/j.nmd.2017.11.004
DO - 10.1016/j.nmd.2017.11.004
M3 - Article
SN - 0960-8966
SP - 197
EP - 207
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
ER -