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 ...
Medscape

Gene Therapy Improves Movement in Older Children With SMA

Determining which patients should receive the treatment will still require individual risk-benefit assessments, experts say.
Kalkine Media

Sarepta Therapeutics (NASDAQ:SRPT) Nasdaq Index Tracks Biotech Advancements

Sarepta Therapeutics (NASDAQ:SRPT) leads in genetic therapies, contributing to ongoing discussions surrounding the nasdaq ...

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.
Clinical Trials Arena on MSN

Sarepta terminates Hansa-partnered gene therapy combination trial

Sarepta and Hansa formed the partnership in 2020 to determine if Idefirix could broaden the patient population for Elevidys.

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 ...
Nasdaq

FDA Restricts Use of Sarepta's Gene Therapy, Adds Safety Warnings

Sarepta Therapeutics SRPT announced that the FDA has approved significant changes to the label of Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). The revised labeling not ...

Sanofi flags second delay to FDA decision on multiple sclerosis drug, shares fall

Shares of French pharmaceutical group Sanofi fell 4% on Monday after it flagged another delay to a U.S. regulatory decision ...