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 ...

Sarepta’s Elevidys Safety Concerns Open Major Opportunity for Solid Biosciences’ (SLDB) SGT-003 in Duchenne Therapy

Solid Biosciences Inc. (NASDAQ:SLDB) is one of the stocks that will double in 2026. On December 4, Needham analyst Gil Blum ...

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 ...

A mom was overjoyed when she learned a drug could save her baby's life. Then she learned it 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.
newscase

Sarepta Therapeutics Engaged in Escalating Patent Dispute

Investor attention has returned to Sarepta Therapeutics amid reports of the biotech firm's vigorous legal strategy to protect ...

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.
STAT

Child’s sudden death unnerves a promising area of gene therapy research

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...