Advancing treatments for rare diseases Together

June 2017

22Jun

Prof Muntoni is the Principal Investigator of the multicenter european phase Ib clinical study in Duchenne Muscular Dystrophy boys. He presented Rimeporide in an oral communication at the 12th European Paediatric Neurology Society Congress in Lyon on June 22nd.

 

On this occasion he gave an update on the preclinical and the clinical development of this innovative treatment that addresses in particular the life-threatening cardiac damage in Duchenne Muscular Dystrophy patients.

16Jun

EspeRare President’s TedMed talk in now online.

In this inspiring speech, Sharon Terry shares her personal story about how, since science didn’t understand her kids’ rare disease, she and her husband decided to study it themselves. Thus they paved the way for patient empowerment in research and drug development.