Pharmaceutical giant Novartis has begun accepting applications for a lottery-based program to give away 100 doses of a gene therapy for spinal muscular atrophy, a sometimes-deadly muscle-wasting ...

The treatment caused muscle cells to begin to divide and the mouse’s ... P400PM_186709); and Fondation Leducq. CITATION: “An Enhancer-Based Gene Therapy Strategy for Spatiotemporal Control of Cargoes ...

It causes relentless muscle breakdown, loss of mobility in early adolescence, and premature death. With no cure currently available, gene therapy has been viewed as one of the most hopeful paths ...

“If you could diagnose CP sooner, you could have better interventions, better physical therapy for ... that circNFIX controlled muscle development by regulating the expression of an important protein ...

More manufacturing muscle looks to be on the way. In 2020, FUJIFILM Diosynth Biotechnologies invested an extra US$120 million in the gene therapy field, including plans for a 6,000-square-metre ...

The one-dose gene therapy – much like rivals from Pfizer and Solid Biosciences – codes for a shortened form of dystrophin that is deficient in patients with the X-linked muscle-wasting ...

A new gene therapy reversed heart failure in pigs by repairing heart function through cBIN1, showing major promise for future ...

At AveXis, a Novartis company, we have built on 30 years of science to deliver on this challenge by demonstrating the long-term curative potential of gene therapy and its capacity to make an ...

the CNIC team developed a gene therapy using adeno-associated viruses (AAV)—a safe delivery system for human patients—to deliver a functional copy of TMEM43 directly into the cardiac muscle ...