Results of Prosensa’s Extended Phase I/II Exon-skipping Trial in Duchenne Muscular Dystrophy Published in the New England Journal of Medicine

Leiden, March 24, 2011 – Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, announces the publication of results from a phase I/II and 12-week extension study of its lead product, PRO051 (GSK2402968) for the treatment of Duchenne Muscular Dystrophy (DMD) in the New England Journal of Medicine (NEJM)[1].

 

Details at

http://www.prosensa.eu/press-release/results-prosensa’s-extended-ph...-

 

Also I saw one more article which has quote from Judith C. van Deutekom, PhD, vice president for discovery at Prosensa Therapeutics,

"We noticed a modest improvement, which is quite remarkable for patients with this disease, in the distance they can walk in six minutes," van Deutekom says. "The dystrophin probably accumulated over time in their muscles and led to the observed improvement."

This is quite encouraging.

Details at

 

http://www.webmd.com/news/20110323/new-muscular-dystropy-treatment-...

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What about the protein found in the urine? Isn't that going to cause problems in the kidney knowing this will be a life long treatment........

http://www.nejm.org/doi/full/10.1056/NEJ...

Extended test results of Prosensa patients (12 weeks at 6mg/kg): "Proteinuria, defined as a protein level above the upper limit of the normal range of 0.15 g per liter, was observed in all 12 patients (mean [±SD] protein level, 0.078±0.038 at baseline and 0.206±0.119 at week 12 of the extension phase)."

I am more excited about

 

Tibialis anterior muscle biopsy was performed at baseline and 2 weeks after the last dose of PRO051 in the 0.5-mg group and at 2 and 7 weeks after the last dose in the three other groups. After an interval of 6 to 15 months after the last dose, each patient restarted treatment at 6.0 mg per kilogram of body weight per week, with close monitoring of safety and clinical-efficacy measures. The current report includes data through 12 weeks of restarted treatment (with biopsy not conducted at 12 weeks). No formal statistical testing was performed, owing to the small number of patients. Data are presented for individual patients and are also summarized.

 

 

Lets wait to get the next set of results.

The results of the 6 minute walk were the most encouraging to me. Instead of a decrease in the distance which usually happens with the disease progression, or even maintaining baseline levels, most of them increased their distance by the end of the extension study.

Three of them went 60+ m past baseline!

There are better details in the NewEngland report

http://www.nejm.org/doi/full/10.1056/NEJMoa1011367#t=abstract

This is the full article for anyone interested.
Attachments:
I don't expect exon skipping to be a life long treatment.  I believe other approaches that are in the pipeline right now just might prove solid, thereby making skipping an option instead of the only item on the menu in our son's future's. 

Ana Vaish said:

What about the protein found in the urine? Isn't that going to cause problems in the kidney knowing this will be a life long treatment........

http://www.nejm.org/doi/full/10.1056/NEJ...

Extended test results of Prosensa patients (12 weeks at 6mg/kg): "Proteinuria, defined as a protein level above the upper limit of the normal range of 0.15 g per liter, was observed in all 12 patients (mean [±SD] protein level, 0.078±0.038 at baseline and 0.206±0.119 at week 12 of the extension phase)."

WebMD has a pretty good article on this as well.

http://www.webmd.com/news/20110323/new-muscular-dystropy-treatment-...

 

Here's a paragraph from the article that got my attention:

Three of the patients improved their walking distance by 213 feet or more. That may not seem like much. But in just the six to 15 months between the first and second phases of the study, these patients' six-minute walking ability had declined by 121 feet.

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