#elevidys
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iclnoticias.com.br
Duas crianças de Porto Alegre receberam infusões de Elevidys para tratamento da distrofia muscular de Duchenne
iclnoticias.com.br
February 28, 2025 at 7:33 PM
pantheradraws.bsky.social
ahead of Sarepta's superior drug); elevidys will be FDA approved on June 21st. To remind you, givinostat is an HDAC inhibitor which will tell my muscles cells to wake up and fix themselves while Elevidys is a mini-gene therapy that will fix my genetic code (the cure itself)....see reply
June 12, 2024 at 11:52 PM
reuters.com
Exclusive: Sarepta says it won't comply with FDA request to stop shipping gene therapy Elevidys reut.rs/4mn74Vd
Exclusive: Sarepta says it won't comply with FDA request to stop shipping gene therapy Elevidys
U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a muscular dystrophy patient who received a different, experimental treatment died, but the company said it would not do so.
reut.rs
July 19, 2025 at 1:40 AM
reuters.com
US FDA adds strongest warning to Sarepta's Elevidys after fatal liver injuries reut.rs/4nVDt5i
US FDA adds strongest warning to Sarepta's Elevidys after fatal liver injuries
The U.S. Food and Drug Administration said on Friday it approved new labeling for Sarepta Therapeutics' gene therapy Elevidys that includes its most serious safety warning and restricts use of the treatment to walking patients with Duchenne muscular dystrophy, or DMD.
reut.rs
November 15, 2025 at 12:45 AM
pantheradraws.bsky.social
Updated Cure News #curepan2024

I'll be starting givinostat on July 10th (if elevidys doesn't come to market first). Right now the two companies that have created these drugs (Italfarmaco & Sarepta, respectively) are racing to reach market first (or rather Italfarmaco is trying to stay... See reply
June 12, 2024 at 11:52 PM
folha.com
Gilmar amplia exceções para ofertar remédio de R$ 18 milhões para distrofia muscular de Duchenne

Suspensão da distribuição do Elevidys não atinge crianças que já completaram sete anos, decide ministro do STF
Elevidys: Gilmar amplia exceções para fornecer remédio - 04/09/2024 - Equilíbrio e Saúde - Folha
Suspensão da distribuição do Elevidys não atinge crianças que já completaram sete anos, decide ministro do STF
mla.bs
September 4, 2024 at 7:22 PM
reuters.com
Exclusive: US FDA to ask Sarepta to stop shipments of gene therapy Elevidys, source says reut.rs/3GxFkhl
Exclusive: US FDA to ask Sarepta to stop shipments of gene therapy Elevidys, source says
The U.S. Food and Drug Administration is planning to ask Sarepta Therapeutics to voluntarily stop all shipments of its gene therapy Elevidys, a source familiar with the matter told Reuters on Friday.
reut.rs
July 18, 2025 at 9:50 PM
reuters.com
US FDA investigates third death after treatment with Sarepta's gene therapy Elevidys reut.rs/46Zx04s
US FDA investigates third death after treatment with Sarepta's gene therapy Elevidys
The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received Sarepta Therapeutics' muscular disorder gene therapy Elevidys.
reut.rs
July 25, 2025 at 10:00 PM
leerezaul24.bsky.social
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
theakarifoundation.bsky.social
Sarepta Therapeutics comparte: 

#TheAkariFoundation #sanantoniotexas #enfermedadesraras #DistrofiaMuscularDeDuchenne #duchenneenespañol #SareptaTherapeutics #Elevidys #AkariSeptiembre
September 30, 2026 at 7:19 PM
reshmagar.bsky.social
I didn't agree with Dr. Marks on every FDA approval decision he made (see Elevidys, Humacyte) but this letter is incredible, reflecting a true public servant and physician who just wanted to make patients' lives better. Thank you for your service, Dr. Marks. cc: @drsforamerica.bsky.social #MedSky
enirenberg.bsky.social Edward Nirenberg @enirenberg.bsky.social · Mar 29
Peter Marks’s resignation letter. Everyone should read this.
Peter Marks, MD, PhD Director, Center for Biologics Evaluation and Research U.S. Food and Drug Administration 10903 New Hampshire Avenue Silver Spring, MD 20903 March 28, 2025 Sara Brenner, MD, MPH Acting Commissioner of Food and Drugs U.S. Food and Drug Administration 10903 New Hampshire Avenue Silver Spring, MD 20903 Dear Dr. Brenner: It is with a heavy heart that I have decided to resign from FDA and retire from federal service as Director of the Center for Biologics Evaluation and Research effective April 5, 2025. I leave behind a staff of professionals who are undoubtedly the most devoted to protecting and promoting the public health of any group of people that I have encountered during my four decades working in the public and private sectors. I have always done my best to advocate for their well-being and I would ask that you do the same during this very difficult time during which their critical importance to the safety and security of our nation may be underappreciated. Over the past years I have been involved in enhancing the safety of our nation's blood supply, in advancing the field of cell and gene therapy, and in responding to public health emergencies. In the last of these, during the COVID-19 pandemic I had the privilege of watching the vision that I conceived for Operation Warp Speed in March 2020 in collaboration with Dr. Robert Kadlec become a reality under the leadership of HHS Secretary Azar and President Trump due to the unwavering commitment of public servants at FDA and elsewhere across the government. At FDA, the tireless efforts of staff across the agency resulted in remarkably expediting the development of vaccines against the virus, meeting the standards for quality, safety, and effectiveness expected by the American public. The vaccines undoubtedly markedly reduced morbidity and mortality from COVID-19 in the United States and elsewhere. Many of these same individuals applied learnings from the pandemic during a flawless response helping… ongoing multistate measles outbreak that is particularly severe in Texas reminds us of what happens when confidence in well-established science underlying public health and well-being is undermined. Measles, which killed more than 100,000 unvaccinated children last year in Africa and Asia owing to pneumonitis and encephalitis caused by the virus, had been eliminated from our shores. The two-dose measles, mumps, rubella vaccine regimen (MMR) using over the past decades has a remarkably favorable benefit-risk profile. The MMR vaccine is 97% or more effective in preventing measles following the two-dose series, and its safety has been remarkably well studied. Though rarely followed by a single fever-related seizure, or very rarely by allergic reactions or blood clotting disorders, the vaccine very simply does not cause autism, nor is it associated with encephalitis or death. It does, however, protect against a potential devasting consequence of prior measles infection, subacute sclerosing panencephalitis (SSPE), which is an untreatable, relentlessly progressive neurologic disorder leading to death in about 1 in 10,000 individuals infected with measles. Undermining confidence in well-established vaccines that have met the high standards for quality, safety, and effectiveness that have been in place for decades at FDA is irresponsible, detrimental to public health, and a clear danger to our nation's health, safety. and security. In the years following the pandemic, at the Center for Biologics Evaluation and Research we have applied the same unwavering commitment to public health priorities to the development of cell and gene therapies to address both hereditary and acquired rare diseases. During my tenure as Center Director we have approved 22 gene therapies, including the first gene therapy ever to be approved in the United States. However, we know that we must do better to expedite the development of treatments for those individual suffering from any one of the thousan…
March 29, 2025 at 3:38 AM
pantheradraws.bsky.social
Cure update: Long story short, Elevidys (by Sarepta) has shown and "robust" improvement in all test patients. The FDA has granted priority review with a target date of June 21st. I could be on the drug by the end of the year. It potentially is the cure. #curepan2024. 🧬💪✨
February 20, 2024 at 6:36 PM
shyl1920.bsky.social
Bir el uzat, bu vatanın evlatları için...
c.org/7CBdFRDRH9
Kampanyayı İmzala
SMA ve DMD hastası çocuklar için Zolgensma ve Elevidys ilaçları ücretsiz olsun
c.org
March 17, 2026 at 8:52 AM
reuters.com
FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy reut.rs/3H4xQSX
FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy
The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received Sarepta Therapeutics' muscular disorder gene therapy Elevidys.
reut.rs
July 25, 2025 at 11:15 PM
folha.com
Elevidys é aplicado no SUS para distrofia de Duchenne
Remédio mais caro do Brasil, de R$ 11 milhões, é aplicado pela primeira vez no SUS
Crianças de Porto Alegre receberam infusões de Elevidys para tratamento da distrofia muscular de Duchenne
www1.folha.uol.com.br
February 21, 2025 at 7:44 PM
statnews.com
Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne muscular dystrophy therapy Elevidys.
www.statnews.com/2025/08/06/s...
Sarepta hired a Trump-connected firm to lobby on Duchenne treatment
Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne muscular dystrophy therapy Elevidys.
www.statnews.com
August 6, 2025 at 5:42 PM
correiocb.bsky.social
Medicamento Elevidys foi utilizado como tratamento da distrofia muscular de Duchenne em duas crianças de Porto Alegre
SUS aplica remédio mais caro do país, de R$ 11 milhões, pela 1ª vez
Medicamento Elevidys foi utilizado como tratamento da distrofia muscular de Duchenne em duas crianças de Porto Alegre
www.correiobraziliense.com.br
February 23, 2025 at 12:12 AM
walkerbragman.bsky.social
In so many ways, Vinay Prasad dug his own grave by trying to appeal to the right.

- He presented himself as a progressive to evangelize their lax pandemic agenda to a wider audience.

- He attacked Elevidys to defend RFK/Makary ousting Peter Marks.

And the leopards ate his face anyway.🤷‍♂️
Vinay Prasad Out At FDA Amid Right-Wing Push
The departure of the contrarian public health official showcases contradictions in the MAHA movement.
www.importantcontext.news
July 30, 2025 at 10:18 PM
abenespina887.bsky.social
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
notdred.bsky.social
Yeah it started with the Elevidys situation where he also made a unilateral call and then Loomer convinced Trump that he was a secret leftist mole. Makary and RFK had to beg Susie Wiles to save his job
February 18, 2026 at 6:18 PM
reuters.com
Sarepta to pause Elevidys gene therapy shipments in US reut.rs/4kNY8GY
Sarepta to pause Elevidys gene therapy shipments in US
Sarepta Therapeutics said on Monday it will pause all shipments of Elevidys in the United States after two teenage boys with a rare condition called Duchenne muscular dystrophy, who had received the company's gene therapy, died of liver failure this year.
reut.rs
July 22, 2025 at 1:50 AM
demetriomota.bsky.social
Fonte: Consultor Jurídico
share.google/ptOcf1ABMrtE....
Registro na Anvisa obriga plano a custear remédio de R$ 16,5 mi
TJ-SP considerou que medicamento Elevidys, usado para Distrofia Muscular de Duchenne (DMD), deve ser fornecido se não houver alternativa
share.google
March 28, 2026 at 12:38 AM
digor77.bsky.social
📱 SOLIDARIEDADE: SALVE O DUDU

Dudu é um garotinho q sofre de Distrofia Muscular de Duchenne. P/ seu tratamento, ele precisa do medicamento (dose única) chamado ELEVIDYS q é bastante caro, por isso, a Família dele precisa do nosso apoio.

Matteus foi convidado p/ ser porta-voz dessa ação solidária:
October 24, 2024 at 3:29 AM
cnn-brasil.bsky.social
Anvisa cancela registro do Elevidys, medicamento para doenças graves
Anvisa cancela registro do Elevidys, medicamento para doenças graves
www.cnnbrasil.com.br
August 25, 2026 at 12:47 AM