PPMD's Blog – November 2017 Archive (7)

Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Duchenne

Solid Biosciences announced today that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne.



The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with Duchenne. Enrollment will begin at the first clinical trial site in the United…

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Added by PPMD on November 30, 2017 at 10:24am — No Comments

Capricor Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for CAP-1002

Capricor Therapeutics today announced that the FDA has cleared its Investigational New Drug (IND) application to conduct a new clinical trial of CAP-1002, its lead investigational therapy, in boys and young men in advanced stages of Duchenne. PPMD looks forward to being join by Capricor for a community webinar tomorrow, November 30 at 1:00 PM EST…

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Added by PPMD on November 29, 2017 at 8:30pm — No Comments

Capricor Reports Significant Improvements in Duchenne Patients Treated with CAP-1002 Investigational Cell Therapy

PPMD is excited to learn that Capricor's investigational cell therapy, CAP-1002, is showing significant improvements in people with Duchenne. We look forward to learning more details about this therapy in a community webinar on Wednesday, November 29 at 1pm eastern. Details coming soon!

Read the release from…

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Added by PPMD on November 15, 2017 at 2:30pm — 1 Comment

PPMD Submits Community Sign-On Letter to FDA Supporting Approval of Ataluren

PPMD has been continuing to engage with both PTC and the FDA on behalf of the patient community regarding the regulatory review of ataluren.



Earlier this month, PPMD reached out to FDA requesting guidance on…

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Added by PPMD on November 13, 2017 at 1:00pm — No Comments

Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne

Wave Life Sciences Ltd. announced the initiation of a global Phase 1 clinical trial for WVE-210201 in Duchenne patients amenable to exon 51 skipping. PPMD is excited by the progress Wave Life Sciences has made in exon skipping. While advances have been made in exon skipping, especially recently, there is more to explore within this technology. We…

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Added by PPMD on November 6, 2017 at 9:09am — No Comments

Sarepta & Nationwide Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne

More good news in gene therapy! Today, Sarepta Therapeutics announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for the micro-dystrophin gene therapy program. The Phase 1/2a clinical trial, now open to enrollment and scheduled for initiation by mid-November, is designed to assess the safety and tolerability of AAVrh74.MHCK7.micro-Dystrophin in individuals…

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Added by PPMD on November 6, 2017 at 8:30am — No Comments

Sarepta Therapeutics & Nationwide Children’s Hospital Announce FDA Clearance of the IND Application for the GALGT2 Gene Therapy Program

Once again, PPMD is excited to see industry, patient groups, and research partnering, as today Nationwide Children's Hospital and Sarepta Therapeutics announced the achievement of an important milestone in…

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Added by PPMD on November 3, 2017 at 8:39am — No Comments

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