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

• Principais autores: Kamornwan Katanyuwong (Autor), Utcharee Intusoma (Autor), Kullasate Sakpichaisakul (Autor), Tanitnun Paprad (Autor), Piradee Suwanpakdee (Autor), Chaiyos Khongkhatithum (Autor), Oranee Sanmaneechai (Autor)
• Formato: Livro
• Publicado em: BMJ Publishing Group, 2024-08-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...