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Spinal muscular atrophy in an upper-middle-income nation before the advent of reimbursed disease-modifying therapies

Objective To elucidate the clinical characteristics and standard of care (SoC) of spinal muscular atrophy (SMA) patients in Thailand, focusing on primary endpoints: age at death and a composite of death or tracheostomy need.Design Retrospective observational study.Setting Seven tertiary centres acro...

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Main Authors: Kamornwan Katanyuwong (Author), Utcharee Intusoma (Author), Kullasate Sakpichaisakul (Author), Tanitnun Paprad (Author), Piradee Suwanpakdee (Author), Chaiyos Khongkhatithum (Author), Oranee Sanmaneechai (Author)
Format: Book
Published: BMJ Publishing Group, 2024-08-01T00:00:00Z.
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