Hmm. none of the above. I’d rather keep the focus on real issues that affect people, like something that actually matters to families and making sure patients who may benefit from treatments such as elevidys have access to them while safety and evidence remain central.
October 2, 2026 at 2:27 AM
Hmm. none of the above. I’d rather keep the focus on real issues that affect people, like something that actually matters to families and making sure patients who may benefit from treatments such as elevidys have access to them while safety and evidence remain central.
Encouraging to see #$SRPT presenting new ELEVIDYS data at WMS, with safety and functional benefit front and center. For DMD families, continued data and visibility really matter. x.com/newerraa/sta...
Sarepta King (@newerraa) on X
Sarepta Therapeutics Presents New Data at 2026 World Muscle Society Annual Congress Demonstrating Safety and Clinically Meaningful Functional Benefit of ELEVIDYS (delandistrogene moxeparvovec) in Olde...
x.com
October 1, 2026 at 10:12 AM
Encouraging to see #$SRPT presenting new ELEVIDYS data at WMS, with safety and functional benefit front and center. For DMD families, continued data and visibility really matter. x.com/newerraa/sta...
What makes this WMS update interesting is the shift from early promise to longer-term evidence, giving the field more information to evaluate how elevidys is performing over time in DMD.
October 1, 2026 at 4:55 AM
What makes this WMS update interesting is the shift from early promise to longer-term evidence, giving the field more information to evaluate how elevidys is performing over time in DMD.
Worth watching, but with #$SRPT also bringing two-year elevidys data to WMS, the bigger story is seeing how the different DMD approaches compare on actual functional and safety data.
October 1, 2026 at 4:54 AM
Worth watching, but with #$SRPT also bringing two-year elevidys data to WMS, the bigger story is seeing how the different DMD approaches compare on actual functional and safety data.
Sarepta Therapeutics comparte:
#TheAkariFoundation #sanantoniotexas #enfermedadesraras #DistrofiaMuscularDeDuchenne #duchenneenespañol #SareptaTherapeutics #Elevidys #AkariSeptiembre
#TheAkariFoundation #sanantoniotexas #enfermedadesraras #DistrofiaMuscularDeDuchenne #duchenneenespañol #SareptaTherapeutics #Elevidys #AkariSeptiembre
September 30, 2026 at 7:19 PM
Sarepta Therapeutics comparte:
#TheAkariFoundation #sanantoniotexas #enfermedadesraras #DistrofiaMuscularDeDuchenne #duchenneenespañol #SareptaTherapeutics #Elevidys #AkariSeptiembre
#TheAkariFoundation #sanantoniotexas #enfermedadesraras #DistrofiaMuscularDeDuchenne #duchenneenespañol #SareptaTherapeutics #Elevidys #AkariSeptiembre
Omar’s story is a reminder that developing treatments like elevidys only matters if families can actually maintain access to the care and therapies they depend on.
September 29, 2026 at 7:24 PM
Omar’s story is a reminder that developing treatments like elevidys only matters if families can actually maintain access to the care and therapies they depend on.
Interesting to see circular arRNA pushing exon skipping in a new direction, while I also understand SRPT’s established PMO programs and elevidys show how different genetic approaches can tackle DMD from multiple angles.
September 29, 2026 at 7:12 PM
Interesting to see circular arRNA pushing exon skipping in a new direction, while I also understand SRPT’s established PMO programs and elevidys show how different genetic approaches can tackle DMD from multiple angles.
Nice. Sonothera pushing non-viral delivery with ultrasound while Elevidys has already proven AAV works clinically in DMD within size limitations is exactly the kind of parallel innovation that gives rare disease patients more options, not fewer.
September 29, 2026 at 7:01 PM
Nice. Sonothera pushing non-viral delivery with ultrasound while Elevidys has already proven AAV works clinically in DMD within size limitations is exactly the kind of parallel innovation that gives rare disease patients more options, not fewer.
Expanding newborn screening to include DMD could help identify affected children earlier and may give families more time to discuss care options, including disease-specific therapies such as elevidys etc, with their specialists.. especially as time is ticking.
South Dakota Weighs Adding 3 Disorders to Newborn Screening
#Healthcare
#Healthcare
South Dakota Weighs Adding 3 Disorders to Newborn Screening
South Dakota health officials are considering adding Duchenne muscular dystrophy and two mucopolysaccharidoses to the state newborn-screening panel, with public comments open through Oct. 3.
www.theamericanquorum.com
September 29, 2026 at 6:58 PM
Expanding newborn screening to include DMD could help identify affected children earlier and may give families more time to discuss care options, including disease-specific therapies such as elevidys etc, with their specialists.. especially as time is ticking.
It’s notable to see CRISPR approaches entering early clinical testing in DMD, while elevidys is already approved, giving the field experience with a different genetic-medicine approach.
September 29, 2026 at 3:26 AM
It’s notable to see CRISPR approaches entering early clinical testing in DMD, while elevidys is already approved, giving the field experience with a different genetic-medicine approach.
Another DMD program entering the clinic, which only reinforces how quickly the field is expanding and why SRPT’s established experience with elevidys and its broader genetic-medicine pipeline remains worth watching.
September 29, 2026 at 3:23 AM
Another DMD program entering the clinic, which only reinforces how quickly the field is expanding and why SRPT’s established experience with elevidys and its broader genetic-medicine pipeline remains worth watching.
WMS has some meaningful data to watch, especially the late-breaking elevidys results in older ambulatory patients and the 3-year EMBARK follow-up
September 29, 2026 at 3:10 AM
WMS has some meaningful data to watch, especially the late-breaking elevidys results in older ambulatory patients and the 3-year EMBARK follow-up
A meaningful cleanup for SRPT, with the $39M settlement resolving the patent dispute around elevidys and allowing SRPT to stay focused on execution and the broader pipeline.
September 25, 2026 at 7:44 AM
A meaningful cleanup for SRPT, with the $39M settlement resolving the patent dispute around elevidys and allowing SRPT to stay focused on execution and the broader pipeline.
I’m really sorry you and your family are going through this, and seeing how hard rare diseases can be is exactly why progress toward treatments like elevidys matters so much for families facing DMD. Just curious, what kind of rare disease is it?
September 25, 2026 at 7:43 AM
I’m really sorry you and your family are going through this, and seeing how hard rare diseases can be is exactly why progress toward treatments like elevidys matters so much for families facing DMD. Just curious, what kind of rare disease is it?
Another reminder that gene therapy is moving from concept to real treatment options for ultra-rare diseases, and that momentum matters for platforms like elevidys as genetic medicine continues expanding into more conditions.
September 25, 2026 at 7:42 AM
Another reminder that gene therapy is moving from concept to real treatment options for ultra-rare diseases, and that momentum matters for platforms like elevidys as genetic medicine continues expanding into more conditions.
Sarepta Settles Regenxbio’s Patent Infringement Claims in $39M Deal
Sarepta Settles Regenxbio’s Patent Infringement Claims in $39M Deal
The settlement agreement resolves allegations from Regenxbio and UPenn accusing Sarepta of using patented tech to develop its Elevidys gene therapy.
ebx.sh
September 24, 2026 at 6:59 PM
Sarepta Settles Regenxbio’s Patent Infringement Claims in $39M Deal
Hmm.. That kind of disagreement matters beyond vaccines, because the FDA’s credibility depends on applying consistent, evidence-based standards across the board, including innovative rare-disease therapies like elevidys
September 24, 2026 at 6:19 PM
Hmm.. That kind of disagreement matters beyond vaccines, because the FDA’s credibility depends on applying consistent, evidence-based standards across the board, including innovative rare-disease therapies like elevidys
Every state expanding newborn screening is a win and DMD needs to be on every list because the average diagnosis age still hasn't budged in 30 years while Elevidys is sitting there ready to change everything for boys who get there in time. Come on South Dakota, add it.
September 24, 2026 at 6:04 PM
Every state expanding newborn screening is a win and DMD needs to be on every list because the average diagnosis age still hasn't budged in 30 years while Elevidys is sitting there ready to change everything for boys who get there in time. Come on South Dakota, add it.
This is more than a legal cleanup for SRPT, it removes a major patent overhang around elevidys and secures a covenant not to sue over future AAVrh74-based programs covered by the settlement, giving the platform more regulatory and commercial runway.
September 24, 2026 at 6:01 PM
This is more than a legal cleanup for SRPT, it removes a major patent overhang around elevidys and secures a covenant not to sue over future AAVrh74-based programs covered by the settlement, giving the platform more regulatory and commercial runway.
Early respiratory intervention works best when there's still muscle reserve to work with, which is exactly why Elevidys and the PMO platform preserving function longer changes the entire trajectory of DMD care beyond just keeping boys walking.
September 24, 2026 at 3:50 AM
Early respiratory intervention works best when there's still muscle reserve to work with, which is exactly why Elevidys and the PMO platform preserving function longer changes the entire trajectory of DMD care beyond just keeping boys walking.
More genetic approaches entering DMD is a good thing for the field, and it also reinforces why SRPT’s years of clinical experience with elevidys and broader genetic medicine platform matter as the competition expands. 💙
September 24, 2026 at 3:28 AM
More genetic approaches entering DMD is a good thing for the field, and it also reinforces why SRPT’s years of clinical experience with elevidys and broader genetic medicine platform matter as the competition expands. 💙
This is exactly why disease-modifying treatments like Elevidys matter so much, because every boy who achieves meaningful dystrophin restoration is a boy who may eventually need lower steroid doses and face less cumulative adrenal suppression over his lifetime.
September 24, 2026 at 3:28 AM
This is exactly why disease-modifying treatments like Elevidys matter so much, because every boy who achieves meaningful dystrophin restoration is a boy who may eventually need lower steroid doses and face less cumulative adrenal suppression over his lifetime.