The parents of two boys born with a rare genetic disorder have demanded the rollout of a life-changing heel prick test – ...

Infants with presymptomatic spinal muscular atrophy who were treated with risdiplam had improved functional and survival outcomes. At 12 months, most treated infants were able to sit and stand ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

Novartis said new interim data from an ongoing phase clinical trial for its $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic ...

Biogen Inc. announced positive interim results from a Phase 1 study of salanersen (BIIB115/ION306), a novel antisense oligonucleotide for treating spinal muscular atrophy (SMA). The data show that ...

Dirty diapers are more than a messy reality of infant care—baby poop can be an indicator of an infant’s gut microbiome and future health. “Three-quarters of babies are at heightened risk of atopic ...

The Walking Egg project is bringing IVF to rural communities in South Africa. This week I’m sending congratulations to two sets of parents in South Africa. Babies Milayah and Rossouw arrived a few ...

Swiss drugmaker Roche on Monday said two-thirds of spinal muscular atrophy (SMA) patients now taking its newly approveddrug Evrysdi have previously received rival treatments Zolgensma from Novartis or ...