Patient support organization Smáci, z.s. associating patients with spinal muscular atrophy

Česká verzia

Authors: V. Hradilek
Authors place of work: Fakulta životního prostředí, Česká zemědělská univerzita, Praha
Published in the journal: Cesk Slov Neurol N 2020; 83/116(Supplementum 2): 45-46
doi: https://doi.org/10.48095/cccsnn20202S45

Summary

Patient organizations (PO) are an important link in the care of patients with rare diseases. These organizations are often associations of patients and their legal representatives who associate on the basis of the current lack of care for these patients. PO SMÁci, z.s. defends the interests of patients with spinal muscular atrophy (SMA) and their families. In 2017, SMÁci helped to promote the treatment of SMA disease in an accelerated process, using a newly registered drug (Spinraza). Until 2017, no medicinal product was registered for SMA under the EMA. At present, patients in the Czech Republic already have 3 treatments available (Spinraza, Zolgensma and Evrisdy). PO SMÁci are in discussion with the stakeholders of the Czech health care system about new treatments which are currently in the development. PO SMÁci promotes the improvement of care for patients with SMA within three main areas, which include patient support, health policy and research.

Keywords:

spinal muscular atrophy – patient organization – health technology assessment – health policy

Zdroje

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