RegulationPriority Review (United States), Fast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Paediatric investigation plan (European Union), Rare Pediatric Disease (United States)

Structure/Sequence

Sequence Code 453698636

Clinical Trials associated with Delandistrogene moxeparvovec

NCT06597656

/ RecruitingPhase 1

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of Delandistrogene Moxeparvovec Following Plasmapheresis in Subjects With Duchenne Muscular Dystrophy and Pre-existing Antibodies to AAVrh74

This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 58 weeks.

Start Date18 Sep 2024

Sponsor / Collaborator

Sarepta Therapeutics, Inc.

NCT06270719

/ RecruitingNot Applicable

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.

Start Date07 Feb 2024

Sponsor / Collaborator

Sarepta Therapeutics, Inc.

NCT06241950

/ Enrolling by invitationPhase 1

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74

This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.

Start Date29 Jan 2024

Sponsor / Collaborator

Sarepta Therapeutics, Inc.

[+1]

100 Clinical Results associated with Delandistrogene moxeparvovec

100 Translational Medicine associated with Delandistrogene moxeparvovec

100 Patents (Medical) associated with Delandistrogene moxeparvovec

Literatures (Medical) associated with Delandistrogene moxeparvovec

10 Jun 2025·NEUROLOGY

Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus

Review

Author: Dowling, James J. ; Rose, Sean C. ; Parsons, Julie A. ; Butterfield, Russell J. ; Puskala Hamel, Katie ; Silsbee, Heather M. ; Caller, Tracie Anne ; Servais, Laurent ; Bharadwaj, Jyoti ; Tolchin, Benjamin ; Oskoui, Maryam

This Evidence in Focus reviews the current evidence on the efficacy and adverse effects of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) and presents clinical considerations regarding use. The author panel systematically reviewed available clinical trial data on delandistrogene moxeparvovec in patients with DMD. The risk of bias was evaluated using the American Academy of Neurology's 2017 therapeutic classification of evidence scheme. Safety information, regulatory decisions, and clinical context were also reviewed. Six clinical trials were identified, of which 4 had peer-reviewed data available. From the 4 studies with available data (2 Class I and 2 Class III), exposure data are available on 134 boys, of which 128 are ambulatory and aged ≥4 to <8 years. Both Class I studies failed to meet the primary functional motor outcome as assessed by change in the North Star Ambulatory Assessment score. Several secondary functional motor outcomes demonstrated improvement in the treatment group with small effect sizes, not meeting statistical significance from hierarchical analysis. Corticosteroid dose exposure was higher in the treatment group in the first 12 weeks after infusion, potentially contributing to measured differences between groups. Safety outcomes were similar across studies with multiple treatment-related adverse events, including peri-infusion effects, immune myositis and myocarditis, thrombocytopenia, and liver toxicity. One death has been reported in an individual who was treated with delandistrogene moxeparvovec outside of a trial. Despite not demonstrating efficacy in its primary outcome, delandistrogene moxeparvovec has been approved by the US Food and Drug Administration (FDA) for use in boys with DMD. This decision was supported by the relative safety of the product and secondary outcome measures data in the phase 3 clinical trial. As the drug may now be actively prescribed in the United States and other countries after FDA approval, providers should be aware of the limitations of the treatment and the need to monitor for immune-related side effects including myocarditis, liver injury, and thrombocytopenia, which may require expanded clinical infrastructure. Additional clinical trials and careful collection of real-world evidence from treated patients will be essential to establish short-term and long-term effectiveness and inform understanding of benefits and risks of delandistrogene moxeparvovec across the lifespan.

04 Mar 2025·Expert Review of Neurotherapeutics

The latest developments in synthetic approaches to Duchenne muscular dystrophy

Review

Author: Johnson, Lucy M. ; Pulskamp, Tariq G. ; Berlau, Daniel J.

INTRODUCTION:

Duchenne muscular dystrophy (DMD) is a rare X-linked genetic disorder caused by mutations in the dystrophin gene, leading to an almost complete absence of dystrophin, which is essential for muscle cell structure and function. This resulting muscle deterioration and fibrosis, eventually causes respiratory failure and cardiomyopathy. While there is currently no cure, existing therapies aim to prolong survival and alleviate symptoms.

AREAS COVERED:

This paper reviews current and emerging therapies for DMD, focusing on their safety and efficacy. Although corticosteroids remain the standard treatment, newly approved drugs such as exon-skipping therapies, vamorolone, delandistrogene moxeparvovec, and givinostat provide new treatment options. Additionally, future therapies, including gene therapy, stem cell treatments, and anti-fibrotic agents, show promise for clinical application.

EXPERT OPINION:

Advancements in DMD treatments have expanded patient options. While gene therapy offers potential for correcting the genetic defect and alleviating symptoms, corticosteroids remain the most cost-effective and well-researched treatment. This is partly due to the lack of compelling long-term safety and efficacy data for gene therapies. The accelerated FDA review process has enabled faster approval of new medications; however many have provided minimal clinical benefit to patients. Despite these challenges, continued drug development and innovative research offer hope to patients.

01 Mar 2025·MOLECULAR THERAPY

A comprehensive atlas of AAV tropism in the mouse

Article

Author: Cox, Aaron R ; Dickinson, Mary E ; Lutz, Cathleen M ; Lagor, William R ; Gaitan, Yaned ; Bulcha, Jote ; Hartig, Sean M ; Chuecos, Marcel A ; Suarez, Carlos Flores ; Gao, Guangping ; Hsu, Chih-Wei ; Wang, Dan ; Snow, Kathy J ; Duryea, Jeff ; Walkey, Christopher J ; Martinez, Alexa E ; Hilsenbeck, Susan G ; Hannigan, Seth ; Mirochnitchenko, Oleg ; De Giorgi, Marco ; Alves-Bezerra, Michele ; Saville, Ethan ; Ljungberg, M Cecilia ; Lanza, Denise G ; Murray, Stephen A ; Heaney, Jason D

Gene therapy with adeno-associated virus (AAV) vectors requires knowledge of their tropism within the body. Here we analyze the tropism of 10 naturally occurring AAV serotypes (AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh8, AAVrh10, and AAVrh74) following systemic delivery into male and female mice. A transgene-expressing ZsGreen and Cre recombinase was used to identify transduction in a cell-dependent manner based on fluorescence. Cre-driven activation of tdTomato fluorescence offered superior sensitivity for transduced cells. All serotypes except AAV3B and AAV4 had high liver tropism. Fluorescence activation revealed transduction of unexpected tissues, including adrenals, testes, and ovaries. Rare transduced cells within tissues were also readily visualized. Biodistribution of AAV genomes correlated with fluorescence, except in immune tissues. AAV4 was found to have a pan-endothelial tropism while also targeting pancreatic beta cells. This public resource enables selection of the best AAV serotypes for basic science and preclinical applications in mice.

267

News (Medical) associated with Delandistrogene moxeparvovec

18 Jun 2025

·www.globenewswire.com

Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients

After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been re-assessed, following two cases of fatal acute liver failure Effective immediately, dosing of non-ambulatory patients, irrespective of age, is paused in the clinical setting; dosing of non-ambulatory patients is discontinued in the commercial setting Roche is working closely with relevant health authorities, investigators and prescribing physicians to ensure they are informed and patient care is being appropriately modified The benefit-risk of Elevidys treatment in ambulatory Duchenne patients remains positive and treatment guidance is unchanged 15 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. In the commercial setting, non-ambulatory patients should no longer receive Elevidys. In the clinical trial setting, enrolment and dosing of non-ambulatory patients will be immediately paused until additional risk mitigation measures (e.g. immune modulatory treatment) are implemented in the study protocol. Health authorities, investigators and physicians are being informed so that patient care can be quickly adjusted. This decision follows careful assessment of two cases in non-ambulatory patients of fatal acute liver failure (ALF), an identified risk of Elevidys and other AAV-mediated gene therapies, which led to a reassessment of the benefit-risk profile as unfavourable for patients with DMD who are non-ambulatory. The new dosing restrictions do not impact the treatment of ambulatory DMD patients of any age, and the benefit-risk ratio remains positive in the ambulatory patient population. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect.” DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. The two fatal ALF cases occurred in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date. Following the first case of fatal ALF, European regulators requested that Roche and Sarepta put temporary clinical holds on Elevidys studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). The temporary clinical holds are still in effect. Outside of Europe, dosing will be paused, effective immediately, for the ENVISION trial. The dosing restrictions will also go into effect for future dosing of commercial non-ambulatory patients. Elevidys has been approved by regulatory authorities in eight Roche territories for the treatment of DMD including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the UAE. In 2019, Roche entered into a global collaboration agreement with Sarepta Therapeutics, Inc. to commercialise Elevidys in territories outside the U.S. Roche and Sarepta jointly manage the clinical studies for Elevidys. Roche is the sponsor of the ENVOL study; Sarepta is the sponsor for all other studies. Elevidys is a one-time treatment administered through a single intravenous dose and the first and only approved gene therapy for Duchenne. It is designed to target the underlying cause of Duchenne by delivering new instructions to cells to produce Elevidys-dystrophin in skeletal, respiratory and cardiac muscles. Elevidys aims to slow the progression of Duchenne by delaying the need for a wheelchair, protecting the heart from damage and a person’s ability to breathe without a respirator for as long as possible. Elevidys uses adeno-associated virus (AAV) vector technology and consists of three parts: a transgene, promoter and vector. Its unique construct optimises delivery to all muscle types, including those of interest for Duchenne treatment. A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is ongoing. To date, more than 900 individuals with Duchenne have been treated with Elevidys through Roche’s clinical development program and in real-world settings. Elevidys has already been approved for the treatment of DMD by 10 regulatory authorities around the world, including the US and Japan. Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics. Duchenne muscular dystrophy (DMD) is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. Average life expectancy is only 28 years. The physical, emotional and financial impact of Duchenne on those affected, their families and caregivers, is profound. Duchenne is an X-linked, rare neuromuscular disease caused by pathogenic variants (mutations) in the DMD gene that disrupt the production of functional dystrophin protein, leading to progressive and irreversible muscle weakness, diminished quality of life and premature death. Dystrophin strengthens and protects muscles and without it, normal activity causes excessive damage to muscle cells as they are more sensitive to injury. Over time, muscle tissue is replaced with scar tissue and fat, causing muscles to weaken. Although Duchenne progresses differently in each individual, its devastating trajectory is well established. Those with Duchenne will eventually lose the ability to use and move their limbs, to breathe on their own and are susceptible to respiratory infections. Muscle damage to the heart causes cardiomyopathy, including rhythm abnormalities and heart failure. Early diagnosis is important for timely intervention to prolong muscle function and preserve quality of life. There is a critical need for disease-modifying treatments that address the underlying cause of DMD before irreversible muscle loss occurs. Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today. Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. The content above comes from the network. if any infringement, please contact us to modify.

Clinical Study

16 Jun 2025

·www.fiercebiotech.com

Sarepta reports 2nd death after DMD gene therapy Elevidys, stops dosing in half of patients

Sarepta Therapeutics and Roche reported the death of a 15-year-old Duchenne muscular dystrophy patient who died after receiving their gene therapy Elevidys in a clinical trial.\n A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile.Sarepta and its ex-U.S. partner Roche reported the death early Sunday. Like the first case, disclosed in March, the patient died after developing acute liver failure (ALF) and was non-ambulatory, meaning the individual was no longer able to walk independently because of the progression of Duchenne.As a result, Sarepta has suspended giving Elevidys to non-ambulatory patients in the commercial setting in the U.S. while the company seeks FDA approval of an enhanced risk mitigation measure involving the immunosuppressant sirolimus to manage liver toxicity.For its part, Roche said it has simply “discontinued” Elevidys in commercial non-ambulatory patients outside the U.S.Meanwhile, Sarepta has paused dosing in a clinical trial called Envision, which is serving as the confirmatory trial required by the FDA for Elevidys’ accelerated approval in non-ambulatory patients. The Massachusetts biotech aims to amend the trial protocol to add the prophylactic use of sirolimus, CEO Doug Ingram said on a conference call Monday.As for Roche, European regulators had previously put three Elevidys trials, including Envision, on temporary clinical holds following the first death report. The latest death was of a 15-year-old in the Envision study, Sarepta’s head of R&D, Louise Rodino-Klapac, Ph.D., said on the call. Without providing details, Rodino-Klapac said there were similarities and differences between the two incidents reported so far. In the first case, the deceased patient also had a recent cytomegalovirus infection, which can infect and damage the liver.“We are currently evaluating this case in the context of this case alone, and then in comparison to the previous case,” Rodino-Klapac said. “We haven’t identified a single risk factor, but we are working expeditiously to identify anything that we could potentially point to in the absence of that.”Liver damage is a classwide risk factor of existing gene therapies based on adeno-associated viral (AAV) vectors. The problem is believed to be related to a T-cell-mediated immune response to the AAV vector causing inflammation of the liver and, in rare cases, acute liver failure.Acute serious liver injury is already listed in the “Warnings and Precautions” section of Elevidys’ label, and Sarepta has been looking to update it after the first death.The two fatal liver failure cases happened after Elevidys was given to more than 900 patients globally, including 140 non-ambulatory patients. While the dosing suspension in the U.S. and proposed risk mitigation method currently only affect non-ambulatory patients, three analysts on Monday’s call questioned whether similar measures should be applied to younger ambulatory patients, too, especially as liver toxicity has been seen in both populations.“We are required to follow the evidence, and right now, the signal that we’ve seen is ALF in the non-ambulatory patient population,” Sarepta CEO Doug Ingram explained on the call.But Ingram acknowledged that an enhanced regimen for ambulatory patients is a valid consideration and that “it’s certainly something that we will evaluate as we go on.” Once the proposed non-ambulatory protocol is in place, physicians will have the option to use sirolimus in ambulatory patients as well, he said.About half of the DMD population is non-ambulatory, according to Ingram. Since Elevidys was initially approved in 2023 to treat ambulatory patients, about 30% of patients who got the one-time gene therapy this year were non-ambulatory, the CEO said.During Monday’s call, Rodino-Klapac presented preclinical data in monkeys showing that sirolimus can lower T cells and suppress the immune response to Elevidys’ AAV vector. The immunosuppressant was also able to control elevations of liver enzymes in monkeys, while expression of the therapeutic gene included in Elevidys was not affected.Sarepta has run the new risk mitigation measures by an expert panel and plans to meet with the FDA soon to discuss the plan, according to Ingram. Because the FDA has proactively asked if Sarepta has considered using additional immunosuppression, including sirolimus, Ingram said he believes the two parties will “have a very constructive discussion.”As the two Sarepta execs noted, sirolimus has been used “pretty commonly” in other gene therapies, albeit in slightly different protocols. The patient deaths mark another turn in Elevidys’ eventful journey. In 2023 and 2024, former director of the FDA’s Center for Biologics Evaluation and Research (CBER), Peter Marks, M.D., Ph.D., overruled the agency’s internal review team twice to grant Elevidys its original accelerated approval in a small patient group and then in a much broader population.The second death has worsened Elevidys’ benefit-risk profile, and the liver toxicity concerns will likely linger and possibly even reach the ambulatory group, Jefferies analyst Andrew Tsai said in a Sunday note. Doctors were initially unfazed by the first patient death but hinted that they would not want to see the death rate increase, as families might then deny treatment rather than merely delaying infusions, Tsai noted.The case also adds uncertainty to Elevidys’ future given that the newly appointed director of CBER, Vinay Prasad, Ph.D., had criticized its approval under Marks. On the bright side, Prasad, along with other U.S. health officials, pledged to facilitate the development of new cell and gene therapies during a roundtable discussion earlier this month.Sarepta’s stock price tumbled nearly 50% on the news as of publication time Monday morning. The company has suspended its revenue guidance, hoping to provide an update in its second-quarter earnings report. The company had recently lowered its total 2025 revenue projection to between $2.3 billion and $2.6 billion from a previous range of up to $3.1 billion, solely because of its lower Elevidys sales outlook.

Gene TherapyClinical StudyAccelerated Approval

16 Jun 2025

·pharma.economictimes.indiatimes.com

Sarepta reports second case of liver failure death after its gene therapy treatment

Bengaluru: Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular dystrophy. The patient was undergoing treatment with Sarepta's therapy called Elevidys , which is the only gene therapy approved by the U.S. Food and Drug Administration for Duchenne muscular dystrophy patients aged four and above. The first instance of death was reported in March this year, a 16-year-old boy who died from acute liver failure months after receiving Elevidys. Liver damage is a known risk with Elevidys and other gene therapies that use adeno-associated viral vectors to infuse modified genes. Sarepta said it is taking steps to mitigate the risk of acute liver failure in patients by working to convene an independent group of experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys. It has also temporarily suspending shipments of the therapy and informed the FDA and global health authorities about the incident. Roche, which partners with Sarepta for commercialization of the gene therapy outside the United States, said in a separate statement on Sunday that it has paused the dosing of Elevidys in patients following the two cases of fatal acute liver failure. (Reporting by Rishabh Jaiswal in Bengaluru; Editing by Lincoln Feast.)

Gene Therapy

100 Deals associated with Delandistrogene moxeparvovec

External Link

KEGG	Wiki	ATC	Drug Bank
-	Delandistrogene moxeparvovec	-	-

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	United States	Sarepta Therapeutics, Inc.	22 Jun 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04626674 (SRP-9001-103 \| ENDEAVOR, Company_Website) Manual	Phase 1	Muscular Dystrophy, Duchenne	13	ELEVIDYS (cohort 6, 2 years old at time of treatment)	cyidmthmel(yykzyjwdph) = nrqrxcduln ubdiulpblc (ksagzwmrut ) View more	Positive	16 May 2025
				ELEVIDYS (cohort 4, 3 years old at time of treatment)	cyidmthmel(yykzyjwdph) = jzddhdtbes ubdiulpblc (ksagzwmrut )
NCT05096221 (EMBARK, Company_Website) Manual	Phase 3	Muscular Dystrophy, Duchenne	14	ELEVIDYS	vmrveortvf(qpbumaiybg) = qrzwsnqcdp uohdoghdeg (ptjrtlbkqv ) View more	Positive	16 May 2025
				external control cohort	-
Delandistrogene Moxeparvovec Clinical Trials (ASGCT2025)	Not Applicable	Muscular Dystrophy, Duchenne Troponin-I (TnI)	156	Delandistrogene Moxeparvovec	yikogbnyjt(wrogybgtob) = At week 52, cardiac MRI performed in a subset of patients from EMBARK revealed no relevant differences in cardiac function (including LVEF), volume, or mass between patients treated with delandistrogene moxeparvovec or placebo rzzqmpjmvw (povxrbjkxu ) View more	Positive	13 May 2025
NCT05096221 (EMBARK \| SRP-9001-301, PipelineReview) Manual	Phase 3	Muscular Dystrophy, Duchenne	125	ELEVIDYS+Placebo (Crossover-Treated Patients)	hvyzjeunms(fnuhxixjwp) = qwcpoampzd jsicwvajoe (evqoggjqey )	Positive	27 Jan 2025
NCT05096221 (EMBARK, Literature) Manual	Phase 3	Muscular Dystrophy, Duchenne	125	delandistrogene moxeparvovec	nyjnipauet(yfjyydzujo) = gtuiofifvb mqbcgpuqbw (buzeudjohi ) View more	Negative	09 Oct 2024
				Placebo	nyjnipauet(yfjyydzujo) = onludekwkq mqbcgpuqbw (buzeudjohi ) View more
NCT05096221 (EMBARK, ASGCT2024)	Phase 3	Muscular Dystrophy, Duchenne	125	Delandistrogene moxeparvovec	zzslxgnfdn(nedpdhvskb): P-Value = 0.24 View more	Positive	07 May 2024
				Delandistrogene Moxeparvovec (Placebo)
NCT04626674 (ENDEAVOR, Pubmed \| Ann Neurol) Manual	Phase 1	Muscular Dystrophy, Duchenne	20	Delandistrogene moxeparvovec	txmzqzvory(xvuvqyfuar) = jbcoaesctu uxqqqyydzk (ybtycqsbpm, 42.6) View more	Positive	01 Nov 2023
NCT05096221 (SRP-9001-301 \| EMBARK, Corporate Publications) Manual	Phase 3	Muscular Dystrophy, Duchenne	125	ELEVIDYS	jnxfqwjpwd(oyumitvhye) = mkwghblzgb ukmhfuvlub (yporrecyhd ) Not Met	Positive	30 Oct 2023
				placebo	jnxfqwjpwd(oyumitvhye) = pchtsejsue ukmhfuvlub (yporrecyhd ) Not Met
FDA Manual	Not Applicable	Muscular Dystrophy, Duchenne	41	ELEVIDYS	hcruonrfvh(wogdadeyim) = Most common adverse reactions across studies (incidence ≥5%) were vomiting and nausea, liver function test increased, pyrexia, and thrombocytopenia. nuqaygpmfo (hkcxbwcbkq ) View more	Positive	22 Jun 2023
				Placebo
NCT03769116 (Study 102, AAN2023)	Phase 2	Muscular Dystrophy, Duchenne	41	Delandistrogene moxeparvovec (SRP-9001)	rsofcewkqv(bmppjmimbs) = The most common treatment-related treatment-emergent adverse events (AEs) were vomiting, decreased appetite and nausea xrhpkcwnhv (ypkwmxmqun )	Positive	25 Apr 2023
				Placebo

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