The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Virginia Tech neuroscientists have uncovered a mitochondrial process that supports the brain cells critical for learning, ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule ...

Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...

Detailed price information for Solid Biosciences Inc (SLDB-Q) from The Globe and Mail including charting and trades.

It is subsequently decomposed by ATP overexpressed in the mitochondria, leading to the release of RES for ATP-blocked metabolic therapy through suppression of the aerobic respiratory chain.

the company’s investigational gene therapy for Leber Hereditary Optic Neuropathy (LHON) due to a mutated ND4 mitochondrial gene. The patients had all participated in the phase III trials RESCUE and ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based ...

It may be, she added, that weekly therapy sessions are simply not scalable to a broad population, and the field should explore light-touch alternatives, like single-session interventions and ...