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Duchenne muscular dystrophy

PharmaDeviceNews Duchenne muscular dystrophy
Signature Biologics’ FDA-cleared enrollment expansion could allow up to 30 additional participants in its investigational UC-MSC program for Duchenne muscular dystrophy, broadening the clinical dataset while safety and efficacy remain under evaluation. Representative image.
Pharma & Biotech

What FDA expanded enrollment means for Signature Biologics’ Duchenne cell therapy program

FDA allows up to 30 more patients in Signature Biologics’ DMD UC-MSC program, expanding access while efficacy remains unproven.

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Greenstone Biosciences is advancing patient-derived cardiac cell research to identify potential therapies targeting cardiac fibrosis and cardiomyopathy in Duchenne muscular dystrophy following an NIH R61 grant. Representative image.
Pharma & Biotech

Can Greenstone Biosciences turn patient-derived heart cells into a Duchenne cardiomyopathy drug?

Greenstone Biosciences secured an NIH R61 grant to discover cardiac fibrosis drugs for Duchenne muscular dystrophy using iPSC and AI models.

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Capricor Therapeutics faces a pivotal FDA decision on deramiocel for Duchenne muscular dystrophy cardiomyopathy, with regulators weighing disputed cardiac efficacy evidence against supportive upper-limb results. Representative image.
Pharma & Biotech

Capricor lost the FDA panel vote 9-3. Why investors have not completely abandoned deramiocel

Capricor Therapeutics faces an August 22 FDA decision for deramiocel after advisers voted that the available evidence did not establish effectiveness in Duchenne muscular dystrophy cardiomyopathy. The disputed case now turns on statistical methods, the treatment indication and whether supportive upper-limb results can overcome unresolved cardiac evidence.

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IPS HEART’s ISX9-CPC investigational cell therapy targets cardiomyopathy associated with muscular dystrophies as the company secures FDA Rare Pediatric Disease Designation, strengthening the regulatory pathway for its preclinical cardiac regeneration program. Representative image.
Pharma & Biotech

IPS HEART has another FDA designation for its heart cell therapy. The bigger test has not started yet

IPS HEART gains FDA rare pediatric designation for ISX9-CPC, but the iPSC heart therapy remains preclinical as human testing becomes the key hurdle.

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Representative image: Clinicians review heart imaging and Duchenne muscular dystrophy trial data as Capricor Therapeutics’ $CAPR deramiocel faces heightened FDA uncertainty following a 9 to 3 advisory committee vote against its cardiomyopathy evidence.
Pharma & Biotech

Conflicting HOPE-3 analyses divide FDA panel over deramiocel in Duchenne muscular dystrophy

FDA advisers voted 9 to 3 against deramiocel’s Duchenne cardiomyopathy evidence after challenging the Phase 3 analysis and trial population.

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Pharma & Biotech

Russell 2000 inclusion puts Precision BioSciences before more investors at a pivotal clinical stage

See how Russell 2000 inclusion expands Precision BioSciences’ investor reach while PBGENE-HBV and PBGENE-DMD face critical clinical tests.

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Representative image: Laboratory research into EXG-7001 highlights Nippon Shinyaku and Elixirgen Therapeutics’ effort to develop a full-length dystrophin mRNA therapy for Duchenne muscular dystrophy.
Pharma & Biotech

Nippon Shinyaku backs EXG-7001 as full-length dystrophin mRNA strategy enters Duchenne race

Discover how Nippon Shinyaku’s EXG-7001 option could advance full-length dystrophin mRNA therapy for Duchenne muscular dystrophy.

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Representative image of a pediatric mobility assessment, highlighting how cTAP’s new Duchenne muscular dystrophy prognostic score could help predict loss of ambulation and improve DMD trial design.
Pharma & Biotech

Could cTAP’s new Duchenne prognostic score change how DMD trials measure mobility risk?

Find out how cTAP’s Duchenne prognostic score could improve DMD trial design and loss of ambulation prediction.

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Representative image of a neuromuscular clinic consultation, reflecting Servier’s acquisition of Edgewise Therapeutics’ sevasemten programme and the growing race to develop oral therapies for Becker and Duchenne muscular dystrophy.
Pharma & Biotech

Can Servier’s $2.65bn Edgewise deal reshape the muscular dystrophy drug race?

Servier is buying sevasemten before pivotal muscular dystrophy data. The deal gives Edgewise cash and investors a sharper cardiology story.

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Representative image: Angelini Pharma’s $4.1 billion Catalyst Pharmaceuticals acquisition highlights the growing race for rare disease and neurology assets, with Firdapse, Agamree and U.S. commercial scale at the center of the deal.
Pharma & Biotech

Angelini Pharma bets $4.1bn on rare neurology scale with Catalyst Pharmaceuticals

Angelini gains U.S. rare disease scale. Catalyst gets certainty, but Firdapse concentration keeps the real test ahead.

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