LYSOGENE

bonjour Descroue,
https://www.raps.org/news-and-articles/news-articles/2020/2/fda-issues-sanfilippo-syndrome-drug-development-gu
(trouvé sur le forum dédié sur bourso),
Ca dit notamment ceci, ce qui semble effectivement très encourageant en terme d'acceptation clinique :
"Due to the rarity of Sanfilippo syndrome, FDA says that a single adequate and well-controlled trial along with confirmatory evidence is sufficient to support traditional approval."
post de tgalet.

Il est mentionné aussi ceci juste après :
"However, in situations where a large treatment effect is not expected, FDA says it “strongly recommends a randomized, parallel group trial design with an appropriate concurrent control group,” noting that sponsors “should use randomization as early as in the first clinical trial involving MPS III patients to allow for maximal use and most efficient use of efficacy data for regulatory purposes.”
Ceci est-il compatible avec le design de l'étude de Lysogène ?
Pas assez connaisseur pour l'appréhender.