Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 euros, and ...

Novartis is on the final straight to approval of its spinal muscular atrophy (SMA) gene therapy Zolgensma in the EU, after getting a recommendation for approval from the CHMP. The positive opinion ...

Panelists discuss how spinal muscular atrophy (SMA) treatment will evolve over the next 5 years, likely incorporating ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns and their families. Now, we’re taking an even earlier step: treating ...

The following story in this series will highlight the first successful use of gene therapy to treat spinal muscular atrophy before birth. The impact is profound for these children and their families.

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that its pivotal dose improves functional outcomes in older boys with the disease.

Leaders from the world of cell and gene therapy, including molecular geneticists, immunotherapists, physicians, nonprofit directors, and patient advocates, shared their personal stories and policy ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and speed of new drug approvals.

The gene therapy technology was licensed by UC San Diego to Eikonoklastes Therapeutics in 2021. Eikonoklastes was granted Orphan Drug Designation (ODD) by the FDA for the use of the patented gene ...