Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy

The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.

The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.

spinal muscular atrophy
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
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
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.