Few fields in medicine are as promising and complex as gene therapy. As researchers race to develop cures for previously ...

The Haslam family knew early on that their 10-year-old and 12-year-old were suffering from muscular dystrophy, but what they didn't know is that one day, the University of New Mexico Hospital would ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

UHealth and Holtz Children’s Hospital physicians are exploring a new gene therapy called ELEVIDYS to treat older Duchenne muscular dystrophy (DMD) patients. The therapy delivers a functional ...

The science has proven that gene therapies are effective treatments, and they have given hope to patients and their families ...

What's on the horizon for the life sciences industry? Robin Johnson, PharmD, Gene Therapy Submissions Lead, inSeption Group What unique challenges do gene therapy submissions face compared to ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

Preliminary 2024 total revenue of $555 million to $560 million, exceeding top end of guidance, including Crysvita® revenue of $405 million to ...

After hours: January 10 at 5:44:16 PM EST Loading Chart for TSHA ...

German regulatory agency, the Paul Ehrlich Institute, granted approval to expand the INVISE Phase 1 clinical trial evaluating INT2104 for the ...

For patients with severe elevations in low-density lipoprotein (LDL) cholesterol (≥190 mg/dL) and a combination of clinical ...

Amid recent backlash stemming from market withdrawals and trial delays, the FDA seeks to further clarify its requirements for ...