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Re: Sarepta updates its rollover study of the 12 boys in their phase II trial with last followup anticipated Feb., 2017bagbest, you are just repeating something you read from another poster. According to the mom, one twin lost ambulation at 24 weeks [6 months on the drug] and the other twin lost ambulation at 48 weeks. Both boys had the lowest baseline on the 6mwt in the trial. Both boys produced dystrophin at 24 and 48 week biopsies. The reasoning from the company says they would have benefited in extending ambulation if they were treated sooner. [biotech5's interview of twin's mom 2/16: ] On the other hand, the numbers from the company's 4 year walk test included in their briefing docs in January, 2016 indicate that a few boys were very close to losing ambulation. The definition of ambulation and walking vary from parent to parent. Some parents in Sarepta various trials have created gofunme pages to help with financial need and some of those sites offer a glimpse into the progression of some boys. From the original 12, the majority of boys entered their 6th year of dosing in August, 2016 and the company hasn't offered any new information from this trial. These are the numbers from the company's presentation almost a year ago. Baseline Yr. 4 Age 30mg/placebo 007 374 197 11.67 008 346 55 14.2 50mg/placebo 30mg 50mg |
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Msg # | Subject | Author | Recs | Date Posted |
12267 | Re: Sarepta updates its rollover study of the 12 boys in their phase II trial with last followup anticipated Feb., 2017 | bioinvest2k | 3 | 12/2/2016 11:19:32 AM |
12268 | Re: Sarepta updates its rollover study of the 12 boys in their phase II trial with last followup anticipated Feb., 2017 | schmiggins | 0 | 12/2/2016 2:54:45 PM |