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On the Hunt for the Next Breakthrough in Motor Neuron Disease
On the Hunt for the Next Breakthrough in Motor Neuron Disease
Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.
Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.
spinal muscular atrophy
Targeting a Genetic Accident to Treat Disease
Deanna MacNeil, PhD | May 9, 2023 | 2 min read
David Liu shares how integrating chemistry and evolution in his research has directed his work on base editing techniques aimed at developing new therapeutics.
Two Children Die After Receiving Novartis Gene Therapy
Andy Carstens | Aug 12, 2022 | 1 min read
No deaths had previously been associated with the muscle-wasting treatment 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.
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.
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.