엑손 스키핑 약 Drisapersen는 일정 수준의 dystrophin이 증가한다고 확인됬습니다.

• 제약 회사 글락소 스미스 클라인 (GSK)는 2 단계, 잠재적으로 dystrophin에 유전자의 엑손 51 대상으로 처리 할 수 ​​있는Duchenne 근육 퇴행 위축 소년을 위한 엑손 - 스킵 약물 drisapersen 비US 재판에서 추가 결과를 발표했다.
• 결과는 연속 또는 간헐적으로 일정량의 drisapersen 주사를받은 시험 참가자는 위약을받은 사람보다 재판의 주 25에서 근육 시료에서 dystrophin에 단백질 수준이 증가한다고나타났습니다.
• 이러한 놀라운 결과는 ​​지속적인 drisapersen으로 처리 된 소년은 위약을받은 사람보다 6 분 상당히 멀리 걸어 보여주는이 같은 시험에서 2013 년 4 월 출시 된 사람들을 추가 할 수 있습니다.
• 더 자세한 dystrophin에 결과는 2013 년 10 월에 예상된다.

Drisapersen에서 보고가 없던 dystrophin양의 증가 소식입니다.

원문나가용.

Multinational pharmaceutical company GlaxoSmithKline (GSK) has released encouraging results about dystrophin protein production in a phase 2, non-U.S. trial of its exon-skipping compound drisapersen in boys with Duchenne muscular dystrophy (DMD).

Dystrophin is the muscle protein missing in DMD; its absence can be caused by any of a number of mutations in the dystrophin gene. Drisapersen (also known as GSK2402968) targets exon 51 of the dystrophin gene and is being developed to treat DMD caused by mutations near this exon. It's one of two exon-skipping drugs for DMD now in clinical trials.

The findings, released in August 2013, supplement trial results released in April that showed participants who received drisapersen every week for 24 weeks ("continuous" treatment group) walked significantly farther in six minutes than those who received a placebo.

The new findings show that, at week 25 of the study, those who received drisapersen had higher dystrophin levels in a muscle sample than those who received a placebo. Specifically:

• Thirteen of the 18 participants (72 percent) who had received drisapersen injections every week — the "continuous" group — showed more dystrophin protein in a muscle sample than they had at the start of the study (baseline).
• Ten of the 17 participants (59 percent) who received injections of drisapersen on a different schedule — the "intermittent" group — showed more dystrophin protein than they had at baseline.
• One child out of 18 (6 percent) in the placebo group showed more dystrophin in his muscles at week 25 than he had at baseline.

Complete results on dystrophin levels in this trial are expected in October 2013, the company has said.

A summary of results available so far for this phase 2, non-U.S. drisapersen study (called DMD114117 by GSK) can be downloaded at Results Summary for 114117.

MDA has been supporting the development of exon skipping as a strategy for the treatment of DMD since the 1990s; for more on the development of this strategy, see DMD: Exon Skipping Timeline.

출처: http://quest.mda.org/news/dmd-exon-skipping-drug-drisapersen-appears-increase-dystrophin-levels