Author Archives: Vanda Pinto

Girl, 16, Becomes 1st SMA Patient to Undergo Hip Replacement Surgery

A surgeon at UC Davis Health has successfully performed the first hip replacement in a person — in this case, a 16-year-old girl — with spinal muscular atrophy (SMA). Before the surgery, the girl, named Malena, had pain and limited movement caused by arthritis that had developed between…

Novartis Boosts Zolgensma Production Capacity After FDA Clearance

Novartis Gene Therapies is expanding its manufacturing capacity for Zolgensma (onasemnogene abeparvovec) — its disease-modifying gene therapy for spinal muscular atrophy (SMA) — after its commercial licensure approval was approved by the U.S. Food and Drug Administration (FDA). The approval will allow the company to produce, test and…

Funding Bolsters Effort to Bring Newborn Screening to Eastern Canada

A CA$414,000 grant was awarded to the Maritimes by Muscular Dystrophy Canada (MDC) and Novartis Pharmaceuticals Canada to further support the implementation of newborn screening for spinal muscular atrophy (SMA). The funding (about $322,600), received by the Maritime Newborn Screening Program (MNSP), will go toward work to…

SadBaby Crowdfunding Aims to Help SMA Families

SadBaby, an organization that raises donations to support children with spinal muscular atrophy (SMA), is launching a crowdfunding platform. The project, based on digital currency donations, intends to gather funds from supporters and investors to help families financially with the costly treatments associated with SMA. “SadBaby is doing…