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Duchenne muscular dystrophy News | Kisho

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Duchenne muscular dystrophy

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Duchenne muscular dystrophy

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rare Duchenne muscular dystrophyTrials & approvals

Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD) - BioSpace

Regulatory & Approvals·2 months ago·Read at source →

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

A study published on PubMed details the clinical, pathological, and genetic characteristics of 23 patients with Duchenne muscular dystrophy (DMD) in northern China. This research contributes to the understanding of DMD's presentation and genetic factors in this specific population.

clinical pathological and genetic characteristics of 23 dmd patients in northern china

PubMed Rare Disease·2 months ago·Read at source →

Original title: “Clinical, pathological, and genetic characteristics of 23 DMD patients in northern China.”

Duchenne muscular dystrophy DMD-related muscular dystrophyTrials & approvals

Grünenthal's tegacorat has received FDA designations for Duchenne muscular dystrophy, positioning it as a potential alternative to glucocorticoid treatments. This development could significantly impact treatment options for patients with this rare disease.

SEGRAM aims to offer an alternative to glucocorticoid‑based treatment

Pharma Times·2 months ago·Read at source →

Original title: “Grünenthal receives FDA designations for tegacorat in Duchenne muscular dystrophy”

Duchenne muscular dystrophyResearch

At BIO, a glimpse behind Lilly and Chai’s partnership

Biotech Business·3 months ago·Read at source →

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

A new study evaluates various natural history modeling methods and their impact on cost-effectiveness decisions in Duchenne muscular dystrophy. This research could influence future economic evaluations and treatment strategies for the disease.

evaluating the impact of different natural history modeling methods on cost effectiveness decisions a case study in duchenne muscular dystrophy

PubMed Rare Disease·3 months ago·Read at source →

Original title: “Evaluating the Impact of Different Natural History Modeling Methods on Cost-Effectiveness Decisions: A Case Study in Duchenne Muscular Dystrophy.”

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

A recent study published in PubMed highlights the prevalence and characteristics of cardiac arrhythmia in patients with Duchenne muscular dystrophy through Holter electrocardiographic monitoring. This research provides valuable insights into the cardiac complications associated with this rare disease.

prevalence and characteristics of cardiac arrhythmia in duchenne muscular dystrophy a retrospective single center holter electrocardiographic monitoring study

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Prevalence and Characteristics of Cardiac Arrhythmia in Duchenne Muscular Dystrophy: A Retrospective, Single-Center Holter Electrocardiographic Monitoring Study.”

Duchenne muscular dystrophyTrials & approvals

Dyne Submits BLA for DMD Exon-Skipping Therapy Z-Rostudirsen and Initiates Phase 3 Trial

Treatment & Clinical News·4 months ago·Read at source →

Duchenne muscular dystrophy myotonic dystrophy type 1+1 moreTrials & approvals

REGENXBIO, Novartis, and Dyne are approaching FDA submissions for treatments targeting muscular dystrophies, specifically Duchenne muscular dystrophy and myotonic dystrophy type 1. This regulatory progress highlights significant advancements in the pipeline for these rare diseases.

Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.

BioSpace·4 months ago·Read at source →

Original title: “REGENXBIO, Novartis, Dyne near FDA submissions in muscular dystrophies”

Duchenne muscular dystrophyResearch

Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission

Treatment & Clinical News·4 months ago·Read at source →

Duchenne muscular dystrophy Becker muscular dystrophyResearch

New research identifies biomarkers linked to the pathological progression of dystrophinopathies, which could enhance patient monitoring and treatment strategies. This discovery may lead to improved clinical outcomes for affected individuals.

identification of biomarkers associated with pathological dynamic progression in dystrophinopathies

PubMed Rare Disease·4 months ago·Read at source →

Original title: “Identification of biomarkers associated with pathological dynamic progression in dystrophinopathies.”

Duchenne muscular dystrophy DMD-related muscular dystrophyTrials & approvals

Entrada Therapeutics' shares plummeted over 50% following disappointing results from a Phase 1/2 study for its Duchenne muscular dystrophy treatment. Analysts noted that the data did not meet expectations, raising concerns about the drug's future prospects.

While the company claimed a victory, analysts said data from a Phase 1/2 study fell short of expectations. Entrada shares subsequently tumbled more than 50%.

BioPharma Dive·5 months ago·Read at source →

Original title: “Entrada shares dive as Duchenne results disappoint”

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

Entrada Therapeutics reported a 2.36% increase in dystrophin levels from its oligonucleotide therapy in early studies, which is below the expected double-digit improvement. This disappointing result may impact future funding and development strategies.

Entrada Therapeutics saw a 2.36% dystrophin increase in patients treated with its oligonucleotide asset—a number that fell short of the company’s prior guidance of a double-digit improvement.

BioSpace·5 months ago·Read at source →

Original title: “Entrada crashes as Duchenne therapy comes in ‘below expectations’ in early study”

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

Research shows that suppression of ANXA11 can restore muscular function in the mdx mouse model of Duchenne muscular dystrophy. This discovery could pave the way for new therapeutic strategies targeting DMD.

anxa11 suppression restores muscular function in the mdx mouse model of duchenne muscular dystrophy dmd

PubMed Rare Disease·5 months ago·Read at source →

Original title: “ANXA11 suppression restores muscular function in the mdx mouse model of Duchenne muscular dystrophy (DMD).”

Duchenne muscular dystrophyFunding

As Duchenne innovation booms, let’s ensure it becomes real progress

BioSpace·5 months ago·Read at source →

Duchenne muscular dystrophyResearch

CAPR Stock Gains Steam: Former Capricor Exec Could Become FDA’s Top Gene Therapy Regulator Ahead Of High-Stakes DMD Decision

Treatment & Clinical News·5 months ago·Read at source →

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

The EVOLVE study provides new insights into the real-world experience of eteplirsen, golodirsen, and casimersen in treating Duchenne muscular dystrophy (DMD). These findings may inform future treatment strategies and patient management.

advancements from the evolve study for assessing real world experience with eteplirsen golodirsen and casimersen for the treatment of dmd

PubMed Rare Disease·5 months ago·Read at source →

Original title: “Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD.”

Duchenne muscular dystrophyResearch

Cure Rare Disease Announces Patient Death in CRISPR Gene Therapy Trial of Duchenne | NeurologyLive - Clinical Neurology News and Neurology Expert Insights

Treatment & Clinical News·6 months ago·Read at source →

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

A recent cost-utility analysis evaluates the treatment of Ataluren combined with standard care versus standard care alone for patients with Duchenne muscular dystrophy in Brazil. This study provides insights into the economic implications of incorporating Ataluren in treatment regimens.

cost utility analysis of the treatment with ataluren plus standard of care compared with standard of care alone in patients with duchenne muscular dystrophy in brazil

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Cost-Utility Analysis of the Treatment With Ataluren Plus Standard of Care Compared With Standard of Care Alone in Patients With Duchenne Muscular Dystrophy in Brazil.”

Duchenne muscular dystrophy DMD-related muscular dystrophyTrials & approvals

Sarepta Therapeutics is preparing to submit a regulatory package to the FDA for Amondys 45 and Vyondys 53, despite a failed confirmatory trial. The outcome of the FDA's review remains uncertain.

Sarepta Therapeutics says the FDA has agreed to review a regulatory package for Amondys 45 and Vyondys 53 after they failed a confirmatory trial, but whether the agency will agree to approve them is still unknown.

BioSpace·6 months ago·Read at source →

Original title: “Sarepta Plans FDA Run for Duchenne Exon Skippers Despite Confirmatory Trial Failure”

Duchenne muscular dystrophy DMD-related muscular dystrophyResearch

A recent study published in PubMed highlights the economic burden of Duchenne muscular dystrophy in Mumbai, India, providing insights into the societal costs associated with the disease. This research underscores the need for targeted interventions and support for affected families.

economic burden of duchenne muscular dystrophy from a societal perspective in mumbai india

PubMed Rare Disease·6 months ago·Read at source →

Original title: “Economic burden of Duchenne muscular dystrophy from a societal perspective in Mumbai, India.”

Previous2 / 4Next

Daily Briefings

Not rare diseases news,news about your rare disease.

Earlier

Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD) - BioSpace

A study published on PubMed details the clinical, pathological, and genetic characteristics of 23 patients with Duchenne muscular dystrophy (DMD) in northern China. This research contributes to the understanding of DMD's presentation and genetic factors in this specific population.

Grünenthal's tegacorat has received FDA designations for Duchenne muscular dystrophy, positioning it as a potential alternative to glucocorticoid treatments. This development could significantly impact treatment options for patients with this rare disease.

At BIO, a glimpse behind Lilly and Chai’s partnership

A new study evaluates various natural history modeling methods and their impact on cost-effectiveness decisions in Duchenne muscular dystrophy. This research could influence future economic evaluations and treatment strategies for the disease.

Dyne Submits BLA for DMD Exon-Skipping Therapy Z-Rostudirsen and Initiates Phase 3 Trial

REGENXBIO, Novartis, and Dyne are approaching FDA submissions for treatments targeting muscular dystrophies, specifically Duchenne muscular dystrophy and myotonic dystrophy type 1. This regulatory progress highlights significant advancements in the pipeline for these rare diseases.

Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission

New research identifies biomarkers linked to the pathological progression of dystrophinopathies, which could enhance patient monitoring and treatment strategies. This discovery may lead to improved clinical outcomes for affected individuals.

Entrada Therapeutics' shares plummeted over 50% following disappointing results from a Phase 1/2 study for its Duchenne muscular dystrophy treatment. Analysts noted that the data did not meet expectations, raising concerns about the drug's future prospects.

Entrada Therapeutics reported a 2.36% increase in dystrophin levels from its oligonucleotide therapy in early studies, which is below the expected double-digit improvement. This disappointing result may impact future funding and development strategies.

Research shows that suppression of ANXA11 can restore muscular function in the mdx mouse model of Duchenne muscular dystrophy. This discovery could pave the way for new therapeutic strategies targeting DMD.

As Duchenne innovation booms, let’s ensure it becomes real progress

CAPR Stock Gains Steam: Former Capricor Exec Could Become FDA’s Top Gene Therapy Regulator Ahead Of High-Stakes DMD Decision

The EVOLVE study provides new insights into the real-world experience of eteplirsen, golodirsen, and casimersen in treating Duchenne muscular dystrophy (DMD). These findings may inform future treatment strategies and patient management.

Cure Rare Disease Announces Patient Death in CRISPR Gene Therapy Trial of Duchenne | NeurologyLive - Clinical Neurology News and Neurology Expert Insights

A recent cost-utility analysis evaluates the treatment of Ataluren combined with standard care versus standard care alone for patients with Duchenne muscular dystrophy in Brazil. This study provides insights into the economic implications of incorporating Ataluren in treatment regimens.

Sarepta Therapeutics is preparing to submit a regulatory package to the FDA for Amondys 45 and Vyondys 53, despite a failed confirmatory trial. The outcome of the FDA's review remains uncertain.

A recent study published in PubMed highlights the economic burden of Duchenne muscular dystrophy in Mumbai, India, providing insights into the societal costs associated with the disease. This research underscores the need for targeted interventions and support for affected families.

Not rare diseases news,
news about your rare disease.