Belgian family raises $2.1M for Zolgensma after Novartis denies compassion use request

A Belgian child is set to receive gene therapy Zolgensma after her parents raised over $2 million for the spinal muscular atrophy treatment. The family appealed for donations after Novartis refused to provide the therapy, which has not been approved in Europe, on a compassionate-use basis.

The De Meyer family have raised €1.8 million ($2 million) through a text messaging campaign and €100,000 through a GoFundMe fundraiser for their 9-month-old girl Pia, who has SMA. They received over 900,000 €2 donations from text messages in the first week of their campaign, Ellen De Meyer, Pia’s mother, told BioCentury...