Building with sign reading “Novartis”
Two Children Die After Receiving Novartis Gene Therapy
No deaths had previously been associated with the muscle-wasting treatment Zolgensma.
Two Children Die After Receiving Novartis Gene Therapy
Two Children Die After Receiving Novartis Gene Therapy

No deaths had previously been associated with the muscle-wasting treatment Zolgensma.

No deaths had previously been associated with the muscle-wasting treatment Zolgensma.

spinal muscular atrophy
Concerns over Efficacy and Cost of Muscle Wasting Treatments
Ruth Williams | Nov 11, 2020
Two new medications for treating a rare and deadly neuromuscular disease have high prices and questionable efficacies, say scientists.
a baby's hand cradled by an adult hand
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lisa Winter | Jan 7, 2020
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
sma spinal muscular atrophy zolgensma in utero fetal gene therapy smn1
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Alejandra Manjarrez | Dec 11, 2019
The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.
FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.