New gene therapy drugs can save lives, but many cost millions

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

What happened when a mom learned a drug to save her baby cost $2 million

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.

Sarepta a new outperform at Wedbush as selloff overdone

Wedbush upgrades Sarepta Therapeutics, citing overdone selloff and potential upside as FDA could re-approval Elevidys for non ...

20 Stocks That Will Double in 2026

Average Upside Potential as of December 10: 104.08% ...
Precision Medicine Online

Dyne Therapeutics Aims to Raise $300M in Public Offering, Prepares to Submit BLA for DMD Drug

The firm plans to seek accelerated approval from the FDA next year, while it launches a Phase III trial to support other regulatory applications.
BioSpace

FDA Greenlights First Gene Therapy for Rare Pediatric Disease

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

FDA approves first gene therapy for rare immune disorder

Dec 9 (Reuters) - The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and ...
ABC7 KABC on MSN

Experimental gene therapy treatment created at UCLA gives 'bubble girl' a new life

An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.
MedPage Today on MSN

Gene Therapy Wins FDA Approval for Life-Threatening Immunodeficiency

In Wiskott-Aldrich syndrome trials, therapy was associated with decreases in bleeding, inf ...