Sarepta Therapeutics comparte a la Comunidad Duchenne:
Nos complace compartir que a inicios de esta semana, los datos de dos años del estudio EMBARK – un estudio pivotal de Fase 3 que evalúa Elevidys – fueron publicados en la revista Neurology and Therapy.
#TheAkariFoundation #SareptaTherapeutics
Nos complace compartir que a inicios de esta semana, los datos de dos años del estudio EMBARK – un estudio pivotal de Fase 3 que evalúa Elevidys – fueron publicados en la revista Neurology and Therapy.
#TheAkariFoundation #SareptaTherapeutics
January 16, 2026 at 6:39 PM
Sarepta Therapeutics comparte a la Comunidad Duchenne:
Nos complace compartir que a inicios de esta semana, los datos de dos años del estudio EMBARK – un estudio pivotal de Fase 3 que evalúa Elevidys – fueron publicados en la revista Neurology and Therapy.
#TheAkariFoundation #SareptaTherapeutics
Nos complace compartir que a inicios de esta semana, los datos de dos años del estudio EMBARK – un estudio pivotal de Fase 3 que evalúa Elevidys – fueron publicados en la revista Neurology and Therapy.
#TheAkariFoundation #SareptaTherapeutics
The drug is currently not administered to non-ambulatory patients due to two deaths linked to liver failure. If the new data shows Elevidys is safe, sales will resume and the stock may gain significant value.
January 14, 2026 at 7:05 PM
The drug is currently not administered to non-ambulatory patients due to two deaths linked to liver failure. If the new data shows Elevidys is safe, sales will resume and the stock may gain significant value.
I believe #SRPT will deliver a big surprise in 2026 when the efficacy data for Elevidys in treating Duchenne muscular dystrophy (DMD) is released.
January 14, 2026 at 7:05 PM
I believe #SRPT will deliver a big surprise in 2026 when the efficacy data for Elevidys in treating Duchenne muscular dystrophy (DMD) is released.
When are you going to wake up and Approve Elevidys for DMD, it is the only treatment that changes lives.
January 14, 2026 at 10:48 AM
When are you going to wake up and Approve Elevidys for DMD, it is the only treatment that changes lives.
Knowing what we know now, I would have designed the trial differently. But when the trial was designed, we did not know what we know now...What can be done to convince me more about the Elevidys treatment effects: show a comparison between placebo and external controls.
January 13, 2026 at 7:44 AM
Knowing what we know now, I would have designed the trial differently. But when the trial was designed, we did not know what we know now...What can be done to convince me more about the Elevidys treatment effects: show a comparison between placebo and external controls.
As Elevidys treatment is accompanied with an increase in steroid dose for at least 90 days, which could have an impact on the function, authors tested the placebo group and saw now improvement in the first year compared to baseline, suggesting no benefit.
January 13, 2026 at 7:33 AM
As Elevidys treatment is accompanied with an increase in steroid dose for at least 90 days, which could have an impact on the function, authors tested the placebo group and saw now improvement in the first year compared to baseline, suggesting no benefit.
There were also improvements for time to rise and 10 meter walk run time. For the 95th percentile of the stride velocity there were no data available from the external controls (it is a new outcome measure). Here Elevidys treated patients were stable for 2 years compared to baseline.
January 13, 2026 at 7:32 AM
There were also improvements for time to rise and 10 meter walk run time. For the 95th percentile of the stride velocity there were no data available from the external controls (it is a new outcome measure). Here Elevidys treated patients were stable for 2 years compared to baseline.
This data is hidden in the supplementary data, but authors justify that it has been shown that the 10 days on/off regimen leads to a worse trajectory than daily steroids (i.e. what all Elevidys treated patients are on), and therefore was (rightfully) excluded.
January 13, 2026 at 7:27 AM
This data is hidden in the supplementary data, but authors justify that it has been shown that the 10 days on/off regimen leads to a worse trajectory than daily steroids (i.e. what all Elevidys treated patients are on), and therefore was (rightfully) excluded.
At the 1 year timepoint there was no significant difference between the primary endpoint (North Star Ambulatory Assessment, NSAA), but there was a trend for improvement Elevidys treated patients.
January 13, 2026 at 7:23 AM
At the 1 year timepoint there was no significant difference between the primary endpoint (North Star Ambulatory Assessment, NSAA), but there was a trend for improvement Elevidys treated patients.
Duchenne muscular dystrophy is caused by lack of dystrophin protein, and Elevidys is a gene addition therapy that aims to restore production of a micro-dystrophin by providing a transgene delivered by adeno-associated viral vector (AAVrh7).
January 13, 2026 at 7:21 AM
Duchenne muscular dystrophy is caused by lack of dystrophin protein, and Elevidys is a gene addition therapy that aims to restore production of a micro-dystrophin by providing a transgene delivered by adeno-associated viral vector (AAVrh7).
#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
#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
The firm's share price fell after a presentation at the conference revealed lower-than-expected Elevidys sales.
Sarepta CEO Signals Intent to Expand Elevidys to Younger Duchenne Patients at JPM
www.precisionmedicineonline.com
January 13, 2026 at 2:57 AM
The firm's share price fell after a presentation at the conference revealed lower-than-expected Elevidys sales.
The SCREENING is $8, but the cost of treatment is much more, and there is no perfect treatment that is fully effective, even Elevidys failed phase III trials. There are also harms from screening and the case for newborn screening for DMD is not a slam-dunk.
December 16, 2025 at 6:46 PM
The SCREENING is $8, but the cost of treatment is much more, and there is no perfect treatment that is fully effective, even Elevidys failed phase III trials. There are also harms from screening and the case for newborn screening for DMD is not a slam-dunk.
A HEARTFELT APPEAL for Meytar Rose, a sweet 8-year-old boy born with Duchenne muscular dystrophy terminal muscle-degenerative disease. Groundbreaking therapy called Elevidys has been developed. Treatment cost is $3.2 million, is not Israel’s health cover. Thank you! www.charidy.com/meitarroz/322
www.charidy.com
December 16, 2025 at 2:29 PM
A HEARTFELT APPEAL for Meytar Rose, a sweet 8-year-old boy born with Duchenne muscular dystrophy terminal muscle-degenerative disease. Groundbreaking therapy called Elevidys has been developed. Treatment cost is $3.2 million, is not Israel’s health cover. Thank you! www.charidy.com/meitarroz/322
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
On December 2, 2025, Sarepta Therapeutics filed three petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to the...
www.jdsupra.com
December 15, 2025 at 10:35 PM
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
On December 2, 2025, Sarepta Therapeutics filed three petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to the...
www.jdsupra.com
December 15, 2025 at 10:35 PM
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
On December 2, 2025, Sarepta Therapeutics filed three petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to the...
www.jdsupra.com
December 15, 2025 at 10:35 PM
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
On December 2, 2025, Sarepta Therapeutics filed three petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to the...
www.jdsupra.com
December 15, 2025 at 10:35 PM
Sarepta Files Three IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
On November 25, 2025, Sarepta Therapeutics filed two petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to the...
www.jdsupra.com
December 9, 2025 at 6:09 PM
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
On November 25, 2025, Sarepta Therapeutics filed two petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to the...
www.jdsupra.com
December 9, 2025 at 6:09 PM
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
On November 25, 2025, Sarepta Therapeutics filed two petitions for inter partes review (“IPR”) challenging Genzyme’s patents relating to the...
www.jdsupra.com
December 9, 2025 at 6:09 PM
Sarepta Files Two IPRs Against Genzyme’s Patents Related to Gene Therapy Elevidys®
This is why elevidys and eteplirsen were approved by FDA, but not by EMA. Authors stress that after approval based on a surrogate endpoint, drug developers still have to collect additional data to confirm efficacy.
December 9, 2025 at 8:47 AM
This is why elevidys and eteplirsen were approved by FDA, but not by EMA. Authors stress that after approval based on a surrogate endpoint, drug developers still have to collect additional data to confirm efficacy.
When reports submitted to FAERS are investigated and used by FDA to take regulatory actions, the agency continues to be opaque about how this data and other sources led to safety actions. FDA recently took significant safety actions for #Elevidys... www.fda.gov/news-events/...
FDA Approves New Safety Warning and Revised Indication that Limits Use for Elevidys Following Reports of Fatal Liver Injury
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 sa...
www.fda.gov
December 8, 2025 at 9:02 PM
When reports submitted to FAERS are investigated and used by FDA to take regulatory actions, the agency continues to be opaque about how this data and other sources led to safety actions. FDA recently took significant safety actions for #Elevidys... www.fda.gov/news-events/...
Steroid doses were increased prior to treatment and then tapered down 3 months after treatment when no adverse events were seen. Authors discuss that longer term follow up for safety and efficiency assessment will be crucial for these and other patients treated with Elevidys.
December 8, 2025 at 12:53 PM
Steroid doses were increased prior to treatment and then tapered down 3 months after treatment when no adverse events were seen. Authors discuss that longer term follow up for safety and efficiency assessment will be crucial for these and other patients treated with Elevidys.
Elevidys is also approved in Qatar and UAE. Here authors report on a case series of 8 Duchenne patients who were treated with Qatar. These patients did not have antibodies against AAV (the virus used to deliver the micro-dystrophin cDNA).
December 8, 2025 at 12:42 PM
Elevidys is also approved in Qatar and UAE. Here authors report on a case series of 8 Duchenne patients who were treated with Qatar. These patients did not have antibodies against AAV (the virus used to deliver the micro-dystrophin cDNA).