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737,961 Results for "sarepta therapeutics inc".
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Duchenne muscular dystrophy
Following Sarepta Woes, Others Look To Unlock the Next Chapter in DMD Treatment
With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.
February 9, 2026
·
11 min read
·
Heather McKenzie
Duchenne muscular dystrophy
Sarepta’s DMD Gene Therapy Staves Off Disease Three Years After Treatment
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
January 26, 2026
·
2 min read
·
Dan Samorodnitsky
Duchenne muscular dystrophy
Sarepta Saga Has ‘Gone on Too Long’ as Competitors Catch Up
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments around the long-time Wall Street darling, and patients may soon begin looking elsewhere.
February 5, 2026
·
5 min read
·
Dan Samorodnitsky
Gene therapy
After Mortality-Marred Year, Sarepta Looks Ahead to Catalyst-Rich 2026
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
January 13, 2026
·
2 min read
·
Tristan Manalac
Podcast
Roche Makes Obesity Splash, Moderna Cans Vaccine Trials, Sarepta’s New Data, More
Roche’s obesity candidate achieves 22.5% weight loss in Phase II; Moderna pulls the plug on late-stage vaccine trials as Health Secretary Robert F. Kennedy’s anti-vaccine policies and rhetoric continues; and embattled gene therapy maker Sarepta announces new data in Duchenne muscular dystrophy.
January 28, 2026
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1 min read
·
Heather McKenzie
Duchenne muscular dystrophy
Sarepta’s DMD Exon-Skippers Fail Confirmatory Study, Stock Craters
Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging trends” in efficacy.
November 4, 2025
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2 min read
·
Tristan Manalac
Regulatory
FDA Imposes Boxed Warning, Narrows Patient Pool for Sarepta’s Duchenne Gene Therapy Elevidys
Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy.
November 17, 2025
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1 min read
·
Tristan Manalac
Gene therapy
Doctors Still Prescribing Sarepta’s DMD Gene Therapy After Safety Concerns
A new analyst survey suggests that doctors are still prescribing Sarepta’s Elevidys, even after a series of deaths in certain populations marred the gene therapy’s record.
September 16, 2025
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3 min read
·
Annalee Armstrong
Gene therapy
Sarepta’s Not Done With Gene Therapy Yet as LGMD Asset Yields Promising Data
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene therapy: its limb-girdle muscular dystrophy candidate. But the treatment’s path forward, analysts say, is highly uncertain.
October 15, 2025
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6 min read
·
Tristan Manalac
Gene therapy
FDA’s Second Platform Designation Goes to Krystal After Sarepta Withdrawal
After the FDA revoked Sarepta Therapeutics’ platform designation in July, Krystal Biotech is “poised to be the first major beneficiary” of this program, according to analysts at William Blair.
October 15, 2025
·
2 min read
·
Tristan Manalac
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