tag:blogger.com,1999:blog-4791085249231790709.post9119393454629175576..comments2023-07-27T05:21:58.602-04:00Comments on Treating Achondroplasia: Vosoritide (BMN-111) induz aumento de 50% na velocidade de crescimento em crianças com acondroplasiaMorrys C. Kaisermannhttp://www.blogger.com/profile/08589139743362708286noreply@blogger.comBlogger6125tag:blogger.com,1999:blog-4791085249231790709.post-62573559480521575342015-09-10T10:02:57.591-04:002015-09-10T10:02:57.591-04:00Hi Wasser, this is one of the million dollar quest...Hi Wasser, this is one of the million dollar questions regarding any therapy for rhizomelic dysplasias such as achondroplasia. In achondroplasia, it is clear that proximal bones are more affected than distal ones, which implies that FGFR3 exerts different levels of activity accross them. How will drugs inhibiting FGFR3 activity work in different bones? Morrys C. Kaisermannhttps://www.blogger.com/profile/08589139743362708286noreply@blogger.comtag:blogger.com,1999:blog-4791085249231790709.post-91424172667418797562015-09-09T08:12:34.581-04:002015-09-09T08:12:34.581-04:00In my opinion, "Changes from baseline in prop...In my opinion, "Changes from baseline in proportionality as measured by upper to lower body ratio were not observed" can be the Achilles heel of the research, but I strongly expect I am wrong. Best regards, Wasserhttps://www.blogger.com/profile/12558693969231064821noreply@blogger.comtag:blogger.com,1999:blog-4791085249231790709.post-38653896018898438312015-06-19T11:55:25.340-04:002015-06-19T11:55:25.340-04:00Olá,
O medicamento ainda está em pesquisa, então o...Olá,<br />O medicamento ainda está em pesquisa, então o que escrevemos aqui é especulativo. Considerando que:<br />1. seja bem sucedido na fase 3, cuja previsão atual é que dure um ano, com início planejado, segundo o desenvolvedor, para o fim deste ano, <br />2. e seja aprovado pela autoridade regulatória americana (FDA)<br />Ele poderia ser lançado nos EUA em 2018. Possivelmente chegaria ao Brasil em 2019.Morrys C. Kaisermannhttps://www.blogger.com/profile/08589139743362708286noreply@blogger.comtag:blogger.com,1999:blog-4791085249231790709.post-20796882361673012682015-06-19T11:40:01.508-04:002015-06-19T11:40:01.508-04:00Gostaria se saber, se já tem alguma previsão de qu...Gostaria se saber, se já tem alguma previsão de quando esse tratamento chegará ao Brasil?? Obrigado!Anonymoushttps://www.blogger.com/profile/06362728435158173691noreply@blogger.comtag:blogger.com,1999:blog-4791085249231790709.post-43236286404927940752015-06-19T09:17:53.906-04:002015-06-19T09:17:53.906-04:00Hi Enrique,
Good question. One of the golden goals...Hi Enrique,<br />Good question. One of the golden goals for therapies for achondroplasia could be set as their ability to restore the body proportions or reduce the member disproportion. The press release describes that they could not see differences in the disproportion at 6 months of therapy. There are several possible explanations for that finding. Let's tackle this question in another future article for the blog. Check the group Achondroplasia in Facebook, where we briefly discuss this topic.<br />Saludos,Morrys C. Kaisermannhttps://www.blogger.com/profile/08589139743362708286noreply@blogger.comtag:blogger.com,1999:blog-4791085249231790709.post-20839907162754510482015-06-18T11:04:18.183-04:002015-06-18T11:04:18.183-04:00Hello, this is a fantastic news, i read all the ar...Hello, this is a fantastic news, i read all the article in the Biomarin web, and i have a question.<br />What is the meaning os this phrase: "Changes from baseline in proportionality as measured by upper to lower body ratio were not observed" ? Google translator does not give a coherent translation in spanish.<br />As always thanksAnonymoushttps://www.blogger.com/profile/05836729309846882687noreply@blogger.com