Phoenix Biotech $sgmo Rare disease: Fabry
biopharmadive.com/news/peter-mar…

$SRPT #Duchenne #GeneTherapy #Elevidys newsdirectory3.com/mom-raises-5-3…

This really gets to the ❤️ of the issue. This video of my son treated with Elevidys was captured this a.m. Ignore the outrageous PJs-socks combo. Don't tell me his improvement with this treatment matters less than your pencil-pushing narrative. Don't.

Relanpejou!!! E por um décimo de segundos houve luz . E o ilustríssimo estava de olho aberto

O ministro do Supremo Tribunal Federal (STF) Alexandre de Moraes assegurou tratamento com Elevidys para Victor Yudi Pecete, de 5 anos, diagnosticado com Distrofia Muscular de Duchenne.

Tomás Ross es un niño de #Coyhaique que fue diagnosticado con distrofia muscular de Duchenne. Su medicamento (Elevidys) tiene un costo de 3.500 millones de pesos y se administra una vez a niños de 4 y 5 años en USA y promete detener o disminuir la progresión de la enfermedad.

JPM:' $SRPT Scenario Table & Upside / Downside Considerations
Related To Elevidys Label Expansion; TP=increased to $177 from $112 prior...'

$SRPT Peter Marks Comment at CureDuchenne Suggests FDA Decision on
Elevidys Label Expansion Unlikely to Occur Before PDUFA Action Date

There is some safety related information from the Elevidys review. Notably perhaps

$LRMR reminds me of Sareptas low dystrophin from Elevidys. Maybe 0.72 is on low end but that could increase over time and could be enough to move the needle.

$SRPT A really engrossing story in Chile - the discussion around these types of therapies is ramping up. #Duchenne #GeneTherapy #Elevidys #RareDisease euro.eseuro.com/health/2555830…

Morgan Stanley Names $SRPT a Catalyst Driven Idea; see an 85% probability of a positive outcome, w/ $SRPT potentially up 27%-50%, on their Base Case #Elevidys Assumption of FDA Label Expansion to Ambulatory Patients of all Ages:

Andrea 🏖 Así lo indican especialistas, organizaciones de la enfermedad por el mundo y la propia marca del medicamento.

duchenne-spain.org/blog/sarepta-t…

elevidys.com

La Subsecretaria está en lo correcto.

Madre de 'Tomás contra Duchenne' alcanza los 3 mil millones de pesos tras su paso por el Biobío.
El objetivo de la caminata de Gómez, es un tratamiento para Tomás. El niño de 5 años padece distrofia muscular de Duchenne, de la que podría recuperarse al acceder al Elevidys.
El

Piper Sandler initiated coverage of $SRPT with an Overweight rating and $157 price target. The analyst likes the prospects for ELEVIDYS to receive an expanded Duchenne muscular dystrophy label on its FDA action date of June 21.

Citi open a Catalyst Watch for $SRPT (Buy/$172) 'on the potential full approval of Elevidys for the treatment of Duchenne muscular dystrophy (DMD) and expansion of the drug label beyond the current restricted 4-5-year-old population by the PDUFA target date of June 21, 2024:'

#apaperaday Today's pick is a letter to the editor from JAMA by Bendicksen et al on the expenditure on approved dystrophin restoring drugs in the USA (elevidys, eteplirsen, casimersen, golodirsen and viltolarsen). DOI: 10.1001/jama.2024.2776

$SRPT Really an incredible story. 32 días de caminata: madre de Tomás Ross se reunió con el presidente Boric en La Moneda | 24 Horas #Duchenne #Elevidys #GeneTherapy #RareDisease #Chile youtube.com/watch?v=L-eD4j…

$SRPT no news yet... But it is very close!

I doubt to see Dr. Marks attending the large CureDuchenne conference on May 24th & no #Elevidys approval yet!

'We’re also honored to include Dr. Marks as our keynote speaker, allowing families to hear the latest on approvals directly

📻 #RADIOCIENCIA | ¿En qué consiste la distrofia muscular de Duchenne y qué tan efectivo es el tratamiento 'Elevidys'? El investigador de la Fundacion Ciencia & Vida y académico de la @usansebastian, Enrique Brandan, explicó los detalles.

cooperativaciencia.cl/radiociencia/2…