zolgensma
Concerns over Efficacy and Cost of Muscle Wasting Treatments
Ruth Williams | Nov 11, 2020 | 5 min read
Two new medications for treating a rare and deadly neuromuscular disease have high prices and questionable efficacies, say scientists.
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Emma Yasinski | Sep 25, 2020 | 5 min read
Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.
Gene Therapy Finds a Fertile Home in Ohio
Shawna Williams | Jan 13, 2020 | 8 min read
The midwestern state has quietly laid the groundwork for a biotech hub.
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lisa Winter | Jan 7, 2020 | 2 min read
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Alejandra Manjarrez, PhD | Dec 11, 2019 | 4 min read
The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.
Leaders of AveXis Out Over Doctored Gene Therapy Data
Kerry Grens | Aug 14, 2019 | 1 min read
Novartis, which bought the biotech firm in 2018, announced that the company’s top scientists have left, and news reports say it’s because they were involved in using manipulated data to get the gene therapy Zolgensma approved.
Some Data Backing Novartis Gene-Therapy Approval Manipulated: FDA
Nicoletta Lanese | Aug 7, 2019 | 2 min read
The agency is now evaluating the implications of the corrupted data behind Zolgensma and whether to “take action” against the pharmaceutical company.
FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019 | 3 min read
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.