@sbkizz Tengo un hijo con Distrofia muscular de Duchenne, enf genetica, el que vive todos los dias la discapacidad progresiva de una enfermedad terminal a edades tempranas sabe sin dudar que daría lo que fuera por que su hijo no tuviese ese gen alterado. No hay nada romántico, es dolor
@endpts@leilei_wuu@Sarepta What is the scientific explanation for the contraindication of exons 8 and 9? What is Sarepta doing about it? I am a doctor and the father of a child with Duchenne, we are excluded from all trials and the possibility of treatment, it is horrible. @Sarepta@endpts $SRPT #duchenne
@buylowsellhighh@douglas_ingram What about the contraindication of exons 8 and 9? Did they say anything about that? What scientific validity does this exclusion have?
Hola @cocoscap comitente 25690, ayer vendí lar ordenes figuran cumplidas pero no me aparece el dinero en portafolio ni poder de compra, ya mande mail y escribí en el chat sin respuestas!!
@JonU235@LifeSciEvents@Sarepta When will mutations at the beginning of the gene be eligible in trials? All children with DMD deserve to have possible treatment. As a doctor and father of a son with DMD I ask companies to think about them. Public evidence for those exclusions is limited.
@ParentProjectMD When will mutations at the beginning of the gene be eligible in trials? All children with DMD deserve to have possible treatment. As a doctor and father of a son with DMD I ask companies to think about them. Public evidence for those exclusions is limited.
@Solid_Bio When will mutations at the beginning of the gene be eligible in trials? All children with DMD deserve to have possible treatment. As a doctor and father of a son with DMD I ask companies to think about them. Public evidence for those exclusions is limited.
@oligogirl I understand that from this case it is modified, but in the study 103 another 4 patients presented elevated troponin without this mutation and it is not considered a contraindication
@oligogirl Then a patient with an 8-9 deletion with generalized weakness was presented. in the setting of a concurrent streptococcal pharyngitis and rhino-enterovirus infection. Elevation of troponin I without compromise of cardiac function.