Emma Yasinski,  —

With Zolgensma, Gene Therapy Takes Big Step Forward in Treating Rare Diseases

Zolgensma‘s approval as the first gene therapy for treating all forms of spinal muscular atrophy (SMA) in children up to 2 years old is another step forward — an exceptional and far-reaching one — in the seeming revolution underway in treating more diseases, and an increasing number of…

Risdiplam Showing Potential to Raise SMN Protein to Healthy Levels in SMA Babies and Adults, PTC Says in Interview

Preliminary data from two ongoing clinical trials into risdiplam (RG7916) — a potential oral therapy for all types of spinal muscular atrophy — support its considerable promise, with treated babies now able to “roll, sit, kick … things that they [otherwise] never do,” the CEO of PTC Therapeutics said in…

AveXis Asking FDA, EU to Approve AVXS-101 as 1st Gene Therapy for SMA Type 1

AveXis has filed a first request for approval of its highly-anticipated gene therapy, AVXS-101, to treat infants with spinal muscular atrophy (SMA) type 1 with the U.S. Food and Drug Administration, CureSMA announced on its website. Similar applications were filed with regulatory authorities in the European Union and Japan, AveXis said in…

Newborn Screening for SMA Key as ‘Every Day Matters,’ Says Mom of Boy with Type 1

When Megan Cardenas and her husband brought their 10-day-old son, Derek, to be screened for spinal muscular atrophy (SMA), the doctor told them their baby was simply lazy. The infant might not be meeting early milestones, but he could roll over when placed on his side — something babies…