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FOUND: 3 - What They Never Told You

FDA Probes Death Of 8-Year-Old Who Received Sarepta's Elevidys

The U.S. Food and Drug Administration (FDA) announced on Friday, after market close, that it is investigating the death of an 8-year-old boy who had received Elevidys, Sarepta Therapeutics Inc.’s (NASDAQ:SRPT) gene therapy for Duchenne muscular dystrophy.

Benzinga

Rocket Refocuses Pipeline As Gene Therapy Sentiment Sours

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a firm focused on developing cardiovascular gene therapy, announced a strategic corporate reorganization to extend its runway.

Benzinga

Sarepta Gets Thumbs Down From Regulators Regarding Elevidys Gene Therapy

On Friday, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).

Benzinga

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Elevidys Halt Sparks Analyst Downgrades—Sarepta Races To Revise Label

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced Monday it will voluntarily and temporarily pause all U.S. shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, effective July 22.

Benzinga

Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that have attracted attention from regulators

The Independent UK

Sarepta Reverses Course, Pauses Elevidys Shipments Amid FDA Safety Review

Benzinga

Sarepta Faces Regulatory Showdown As Analysts Cut Price Targets

On Friday, the U.S. Food and Drug Administration placed Sarepta Therapeutics Inc.’s (NASDAQ:SRPT) investigational gene therapy clinical trials for limb girdle muscular dystrophy (LGMD) on a clinical hold.

Benzinga

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Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys

The ground shook for Sarepta Therapeutics, Inc. (NASDAQ:SRPT) last week as the U.S. Food and Drug Administration (FDA), alarmed by a third patient death tied to its gene therapy platform, pulled a rare move: requesting a voluntary halt to Elevidys shipments. The company’s response? A resounding “no,” igniting a major…

Benzinga

Gene therapy maker Sarepta tells FDA it won't halt shipments despite patient deaths

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene therapy

The Independent UK

Sarepta's Setback Deepens With Another Gene Therapy Fatality

Sarepta Therapeutics (NASDAQ:SRPT) shares plummeted on Friday following reports of a third patient death linked to its gene therapy pipeline this year, further clouding investor confidence despite the company’s recent efforts to provide clarity on its restructuring and FDA label plans.

Benzinga

Sarepta Therapeutics Stock Plunges Over 30% In Friday Pre-Market: What's Going On

Shares of Sarepta Therapeutics (NASDAQ:SRPT) plunged over 30% in Friday pre-market after a clinical trial participant died following treatment with a gene therapy, marking the third death linked to the company’s experimental treatments.

Benzinga

Sarepta Therapeutics' Reorganization Boosts Investor Confidence

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stock is trading higher on Thursday after the company issued an after-hours update on ongoing label expansion efforts for Elevidys, its approved gene therapy for Duchenne muscular dystrophy.

Benzinga

Breakthrough gene therapy jab reverses hearing loss in weeks

Researcher says this type of treatment for deafness is ‘just the beginning’

The Independent UK

Gene therapy restores hearing in toddlers and teenagers born with congenital deafness – new research

Up to three in every 1,000 newborns has hearing loss in one or both ears. While cochlear implants offer remarkable hope for these children, it requires invasive surgery. These implants also cannot fully replicate the nuance of natural hearing.

The Conversation

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The Independent UK

Three-year-old becomes youngest patient to receive groundbreaking gene therapy

IN FULL

The Independent UK

Mother’s delight as gene therapy offers hope to three-year-old

Gunreet Kaur has a rare inherited condition which can be fatal.

The Independent UK

Girl, 3, becomes youngest patient to receive groundbreaking treatment

It is the first NHS England commissioned gene therapy in the UK infused directly into the brain

Benzinga

Fractyl Health Stock Is Trading Lower Monday: What's Going On?

Fractyl Health, Inc. (NASDAQ:GUTS) shares are trading lower. The company announced new preclinical data from its Rejuva Smart GLP-1 pancreatic gene therapy platform.

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The Independent UK

Second patient death reported with gene therapy for muscular dystrophy

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene therapy for muscular dystrophy

The Independent UK

Boy with ‘death sentence’ disease living a normal life after breakthrough gene therapy

Four-year-old Eisa can now play football and go to school – things his family feared he may never do

Evening Standard

London hospital gene therapy breakthrough gives boy with ‘death sentence’ disease a normal life

Eisa Hussein took part in a clinical trial testing the new gene therapy at Great Ormond Street Hospital

MarketBeat

Pharma Frenzy: Volatility Ignites Biotech Sector

Two biotech companies have surged on recent developments, illustrating biotech's volatility and prompting investors to evaluate potential long-term value.

The Independent UK

Breakthrough for baby with rare illness in world-first gene therapy trial

Fourteen-month-old Tomas was diagnosed with ornithine transcarbamylase (OTC) deficiency when he was a few weeks old

The Independent UK

Baby given new gene therapy in groundbreaking trial

It is hoped that the new therapy will work as well as a liver transplant for some patients.

StockNews.com

3 Gene Therapy Stocks With Game-Changing Potential

Gene therapy stocks are transforming healthcare with revolutionary treatments. Therefore, as this sector continues to evolve, investing in gene therapy stocks like Pfizer (PFE), Sanofi (SNY), and Novartis AG (NVS) could be beneficial. Read on….

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