#delandistrogene
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rarediseaseadvisor.bsky.social
@sarepta.com announced positive 3-year functional outcomes from the #EMBARK study evaluating delandistrogene moxeparvovec-rokl in ambulatory patients with Duchenne muscular dystrophy (#DMD).

Learn more: https://bit.ly/4qLboQH

#RareDisease #DuchenneMuscularDystrophy #MedSky
Three-Year Results Show Greater Slowing of Progression With Elevidys® for Ambulatory DMD
Sarepta announced the results of year 3 of the EMBARK trial, showing sustained functional benefits of delandistrogene moxeparvovec in DMD.
bit.ly
January 27, 2026 at 7:12 PM
oligogirl.bsky.social
#apaperaday Today's pick is hot of the press: the 2 year analysis of EMBARK study with delandistrogene moxeparvovec (aka Elevidys) in ambulatory Duchenne patients. Already presented at conferences, now out as a peer reviewed paper in Neurology Therapy by Mendell et al DOI: 10.1007/s40120-025-00879-8
January 13, 2026 at 7:18 AM
mdpakistanorg.bsky.social
Sarepta Therapeutics, Inc. announced that the FDA has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). Purpose of Cohort 8 is evaluate use of enhanced immunosuppressive regimen part of treatment with ELEVIDYS (delandistrogene moxeparvovec-rokl) for non-ambulant individuals with DMD.
November 26, 2025 at 7:19 AM
theakarifoundation.bsky.social
El propósito de la Cohorte 8 es evaluar el uso de un régimen inmunosupresor mejorado como parte del tratamiento con ELEVIDYS (delandistrogene-moxeparvovec) para personas no ambulatorias que tienen distrofia muscular de Duchenne.

🔗Lee el artículo completo en el siguiente link:
lnkd.in/eRzmdhhH
November 26, 2025 at 2:02 AM
medpagetoday.com
The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and changed the indication of the drug after reports of fatal liver injury emerged, the agency announced Friday.
https://www.medpagetoday.com/neurology/generalneurology/118544
November 18, 2025 at 7:20 PM
theakarifoundation.bsky.social
Sarepta Therapeutics nos comparte lo que ha cambiado con la actualización de la información de la receta (también conocida como “la etiqueta”) de ELEVIDYS (delandistrogene moxeparvovec-rokl), la única terapia aprobada para la distrofia muscular de Duchenne.

#TheAkariFoundation #SareptaTherapeutics
November 17, 2025 at 4:36 PM
fda-biologics-bot.bsky.social
Description: The U.S. Food and Drug Administration today announced it is taking action to approve new labeling submitted by the company that includes the addition of a Boxed Warning, the agency’s most prominent safety warning, to Elevidys (delandistrogene moxeparvovec-rokl), and that the indication
November 15, 2025 at 6:00 AM
oligoclonalband.medsky.social
Most exciting, FDA-approval of delandistrogene moxeparvovec-rokl (whew): delivers a microdystrophin to the cells. Not a cure, but decreases muscle degeneration.
Requires immunosuppressant therapy. Must be monitored for liver injury - get other specialists involved
October 11, 2025 at 9:27 PM
some-news.bsky.social
EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its...
Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
www.roche.com
July 27, 2025 at 7:59 AM
theakarifoundation.bsky.social
La (FDA, por sus siglas en inglés) emitió un comunicado de prensa anunciando una investigación sobre el fallecimiento de un paciente de ocho años con Distrofia
Muscular de Duchenne (Duchenne) que había recibido la terapia génica ELEVIDYS (delandistrogene moxeparvovec).
July 26, 2025 at 3:03 PM
dmdwarrior.com
FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys

#dmd #sarepta #roche #elevidys #fda #duchenne

👉 Read More: DMDWarrioR.com
FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and voluntary suspension of product distribution as it investigates the safety concerns. The U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy.
July 26, 2025 at 1:55 PM
dmdwarrior.com
The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys

#dmd #sarepta #roche #elevidys #europe #ema

👉 Read More: DMDWarrioR.com
The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys A European Medicines Agency committee ruled that the therapy, called Elevidys, failed to show in studies that it improved patients’ movement abilities. Roche announced today that 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).
July 25, 2025 at 12:03 PM
theakarifoundation.bsky.social
La semana pasada, la FDA solicitó que Sarepta detuviera voluntariamente los envíos de delandistrogene moxeparvovec en EE. UU. Hemos estado trabajando con urgencia para determinar qué medida tomar en los territorios Roche fuera de EE. UU.
July 24, 2025 at 4:59 PM
medpagetoday.com
Sarepta Therapeutics will continue to ship its gene therapy delandistrogene moxeparvovec (Elevidys) to ambulant patients with Duchenne muscular dystrophy, despite the FDA's request to halt distribution.
www.medpagetoday.com/neurology/ge...
Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths
Sarepta says it will continue to ship Elevidys to ambulant Duchenne patients
www.medpagetoday.com
July 21, 2025 at 6:59 PM
iamapyriteking.bsky.social
"Elevidys delandistrogene moxeparvovec"? Sounds like they were killed by three characters from a terrible fantasy novel...
July 19, 2025 at 12:36 PM
medpagetoday.com
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy, will carry a black box warning for acute liver injury and acute liver failure.
www.medpagetoday.com/neurology/ge...
Duchenne Gene Therapy Will Undergo Changes After Patient Deaths
Elevidys will carry a black box warning
www.medpagetoday.com
July 18, 2025 at 1:25 PM
castltrastondrs.bsky.social
such as #aducanumab for #Alzheimerdisease, #tofersen for ALS , #delandistrogene #moxeparvovec for #Duchennemusculardystrophy & #voxelotor for #sicklecelldisease
@jama.com

ja.ma/3GHGhDE

jamanetwork.com/journals/jam...
Regulatory and Clinical Outcomes of Nononcology Accelerated Approvals
This study uses US Food and Drug Administration (FDA) data to characterize pivotal and confirmatory trials supporting agency decision-making in the context of the FDA accelerated approval pathway for ...
jamanetwork.com
July 14, 2025 at 4:03 PM
medpagetoday.com
The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with delandistrogene moxeparvovec (Elevidys) gene therapy.
www.medpagetoday.com/neurology/ge...
FDA Probes Gene Therapy After Two Deaths
Investigation focuses on liver failure after Elevidys treatment for Duchenne muscular dystrophy
www.medpagetoday.com
June 25, 2025 at 7:56 PM
pennmedcso.bsky.social
In @GreenJournal, James Dowling (@PennNeurology/ @penngenetics.bsky.social) & colleagues review the current evidence on the efficacy & adverse effects of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy & present clinical use considerations www.neurology.org/doi/10.1212/...
June 2, 2025 at 1:28 PM
oligogirl.bsky.social
#apaperaday First post-holiday Duchenne related paper is a report of American Association of Neurology Guidelines Subcommittee by Oskoui et al on evidence for delandistrogene moxeparvovec (elevidys) for Duchenne muscular dystrophy patients published in Neurology. doi: 10.1212/WNL.0000000000213604
May 29, 2025 at 8:31 AM
jonu235.bsky.social
Life-Changing New Gene Therapy For #Duchenne Muscular Dystrophy: "children who have received the medication have been able to stay active and the effects of the one time infusion do not seem to wear off" 😲💪 #Elevidys delandistrogene moxeparvovec @sarepta.com www.texaschildrens.org/content/well...
Life-Changing New Gene Therapy For Duchenne Muscular Dystrophy | Texas Children's
Many chronic illnesses of childhood have a genetic cause. The correct genetic code is necessary to make functional proteins that do important work in the body, such as making red blood cells or hormon...
www.texaschildrens.org
May 21, 2025 at 4:38 PM
jonu235.bsky.social
"Statistically significant differences were observed on all key endpoints; Significant functional benefits for 8 and 9-year-olds with Duchenne in Part 2 of the EMBARK study" #Elevidys delandistrogene moxeparvovec #Duchenne muscular dystrophy #GeneTherapy investorrelations.sarepta.com/news-release...
Sarepta Therapeutics Presents Data at the American Society of Gene & Cell Therapy Conference, Including Statistically Significant Functional Outcomes for 8- and 9-Year-Old Patients in New Data Analysis of EMBARK Part 2 05/16/25 9:00 AM EDT Significant functional benefits for 8- and 9-year-olds with Duchenne in Part 2 of the EMBARK study, contributing to the evidence of stabilization or slowing of disease progression in later childhood when muscle weakness typically progresses Statistically significant differences were observed on all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort In the recent analysis of Part 2 of the EMBARK study, participants with Duchenne muscular dystrophy who had received a placebo in Part 1 and were aged 8 to 9 years (n=14) at crossover were included. At one year post ELEVIDYS treatment, there were between-group differences (least square means) on all key endpoints that were statistically significant, including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort. "The latest data from the EMBARK study highlighting motor function improvements in 8- and 9-year-old boys is encouraging and adds to the growing body of evidence supporting ELEVIDYS," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics at the University of Arkansas for Medical Sciences and Arkansas Children’s Hospital. "What stands out is that these patients were treated at an age when motor decline is typically expected in those with Duchenne. Yet, those who received ELEVIDYS demonstrated statistically significant and clinically meaningful functional improvements compared to external controls." The results presented at ASGCT are from the ongoing analysis of results from Part 2 of EMBARK, which compared two-year outcomes from 63 participants against data from an external control group of untreated individuals with Duchenne. Results at two years post-treatment showed that individuals treated with ELEVIDYS had better outcomes in multiple motor function measures, compared to a well-matched external control group. Additionally, no new safety signals were observed in the EMBARK study over the two-year duration and, in a subset of patients (n=16), micro-dystrophin expression and sarcolemmal localization was sustained from Week 12 to Week 64. “This has been a significant year for our neuromuscular portfolio, with multiple, ongoing analyses and longer-term data on efficacy and safety presented for ELEVIDYS,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. “Building on the topline EMBARK Part 2 data from earlier this year, we’re committed to sharing ongoing analyses as fast as possible. The one-year results of patients treated with ELEVIDYS at 8 to 9 years old provide evidence that those treated with gene therapy outperform those who don’t receive it at a critical point when more dramatic functional decline is expected.”
May 16, 2025 at 4:31 PM
jonu235.bsky.social
Protein sure is a hot topic these days! "Treatment with #ELEVIDYS (delandistrogene moxeparvovec) for #Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6)" 💪💪💪 #GeneTherapy investorrelations.sarepta.com/news-release...
Sarepta Therapeutics Shares New Protein Expression and Safety Results from ENDEAVOR in Participants 2 Years Old at Time of Treatment - Treatment with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6) - Safety profile consistent with prior studies of ELEVIDYS and real-world experience CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 16, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Treatment with ELEVIDYS in the ENDEAVOR participants in cohort 6 who were 2 years old at the time of treatment (n=6), demonstrated mean expression of 93.87% of normal, as measured by western blot, and 79.9 percent dystrophin positive fibers (PDPF), as measured by immunofluorescence. The results were seen in biopsies taken 12 weeks after treatment. Safety in cohort 6 was consistent with clinical and real-world experience with ELEVIDYS. The most common adverse events were nausea and vomiting. Elevated liver enzymes were seen in two patients and resolved with steroid administration. “The strength of the biomarker results that we are seeing in younger patients is extremely encouraging and we have a meeting with U.S. FDA next month to discuss expanding the ELEVIDYS label to include younger patients,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. “In addition to positive expression results, the safety profile in these patients is consistent with what we’ve seen in prior studies and in patients who have been prescribed treatment.” Sarepta had previously shared expression and safety from Cohort 4 of ENDEAVOR, in which participants were 3 years old at the time of treatment. In biopsies taken 12 weeks after treatment with ELEVIDYS, participants in Cohort 4 (n=7) demonstrated mean protein levels of 99.64 percent, as measured by western blot. More than 25 patients under the age of 4 have now been treated in our clinical studies.
May 16, 2025 at 3:57 PM
jonu235.bsky.social
Chugai Receives Regulatory Approval for #Elevidys (delandistrogene moxeparvovec) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan 💪💚https://www.chugai-pharm.co.jp/english/news/detail/20250513181500_1160.html
Approval Information Product Name: ELEVIDYS® Intravenous Infusion Generic Name: delandistrogene moxeparvovec Contraindication and Prohibition (partial excerpt): Patients do not have a deletion of any portion or the entirety of exon 8 and/or exon 9 in the dystrophin gene Efficacy or Effects: Duchenne muscular dystrophy Indicated for patients who meet all of the following criteria: Patients who are negative for anti-AAVrh74 antibodies Ambulatory patients Patients aged 3 years or older and younger than 8 years Approval Conditions and Time Limit: [Approval Conditions] During the period until the reapplication for marketing approval of this product after conditional and time-limited approval, post-marketing approval condition evaluation shall be conducted through clinical trials aimed at confirming the long-term efficacy and safety of this product, as well as post-marketing surveillance targeting all cases in which this product is used. Necessary measures shall be taken, including dissemination of proper use guidelines developed in cooperation with relevant academic societies, to ensure that physicians with sufficient knowledge and experience in Duchenne muscular dystrophy use this product in accordance with the "Efficacy or Effects" and "Dosage and Administration" after thoroughly acquiring knowledge of the clinical trial results and adverse events of this product, at medical institutions with established systems for treating Duchenne muscular dystrophy. Necessary measures shall be taken, including dissemination of the usage regulations, to ensure that this product is used in compliance with the Type 1 Use regulations approved under the "Act on the Conservation and Sustainable Use of Biological Diversity through Regulations on the Use of Living Modified Organisms (Act No. 97 of 2003)." [Time Limit] 3 years [Reference] Chugai Files Elevidys (SRP-9001) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan (Press release by Chugai issued on August 14, 2024) https://www.chugai-pharm.co.jp/english/news/detail/20240814150000_1091.html Roche Announces Outcomes for EMBARK Study of Elevidys in Duchenne Muscular Dystrophy (Press release by Chugai issued on October 31, 2023)
May 13, 2025 at 5:07 PM
jonu235.bsky.social
How Gene Therapy Is Changing Logan’s Life #Duchenne muscular dystrophy #RareDisease #Elevidys delandistrogene moxeparvovec #GeneTherapy
www.ivanhoe.com/family-healt...
How Gene Therapy Is Changing Logan’s Life - Ivanhoe Broadcast News, Inc.
Duchenne Muscular Dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe, and live independently. But now there’s a b...
www.ivanhoe.com
May 5, 2025 at 7:12 PM