SMA-EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe

Eugenio Maria Mercuri, Nathalie Kayadjanian, Arthur Burghes, Richard S Finkel, Francoise Rouault, Inge Schwersenz, Kevin Talbot

Risultato della ricerca: Contributo in rivistaArticolo in rivista

16 Citazioni (Scopus)

Abstract

Spinal muscular atrophy (SMA) is the most common lethal recessive disease in childhood, and there is currently no effective treatment to halt disease progression. The translation of scientific advances into effective therapies is hampered by major roadblocks in clinical trials, including the complex regulatory environment in Europe, variations in standards of care, patient ascertainment and enrolment, a narrow therapeutic window and a lack of biomarkers of efficacy. In this context, SMA-Europe organized its first international workshop in July 2012 in Rome, gathering 34 scientists, clinicians and representatives of patient organizations to establish recommendations for improving clinical trials for SMAa.
Lingua originaleEnglish
pagine (da-a)44-44
Numero di pagine1
RivistaOrphanet Journal of Rare Diseases
Volume8
DOI
Stato di pubblicazionePubblicato - 2013

Keywords

  • Clinical Trials as Topic
  • Europe
  • Humans
  • Muscular Atrophy, Spinal

Fingerprint Entra nei temi di ricerca di 'SMA-EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe'. Insieme formano una fingerprint unica.

Cita questo