Ahulken compounds

Ahulken compounds: novel calcium regulators for Duchenne muscular dystrophy

Ainara Vallejo
University of the Basque Country – IIS Biodonostia

July 2018

This is a proposal from Dr Vallejo and colleagues to slow down the progression of Duchenne muscular dystrophy (DMD) with new chemical entities, known as Ahulken compounds (AHK). This proposal is based on successful pilot studies that indicate these compounds regulate calcium in vitro in dystrophin-deficient human myotubes. In vivo pilot studies have also shown improvements in muscle function in mice.

The committee welcomed the opportunity to review this application at an early stage of development and recognises that this is a possible way to reduce disease progression. TACT suggests more preclinical studies to confirm the compound’s safety and superiority. The report makes further recommendations on performing independent validation of mdx studies in collaboration with experts in the DMD field.

17 Dec 2018