PPMD's Blog – May 2014 Archive (7)

PTC Therapeutics Receives Positive Opinion from CHMP for Translarna (Ataluren)

Read the press release from PTC Therapeutics:



The first treatment for the underlying cause of Duchenne muscular dystrophy

SOUTH PLAINFIELD, NJ – May 23, 2014 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for…

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Added by PPMD on May 23, 2014 at 9:30am — No Comments

Drisapersen Regulatory Discussions Ongoing & Re-Dosing on Track

Below you can find the latest update from Prosensa regarding a potential regulatory path forward for drisapersen.







Regulatory discussions ongoing & re-dosing on track

Dear Patient Group…

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Added by PPMD on May 22, 2014 at 8:00pm — No Comments

Catena®/Raxone® Positive Results Reported from Phase III Duchenne Trial

Santhera reported positive results today from Catena®/Raxone® in their Phase III Duchenne trial. These results are supported by additional respiratory function data. By including pulmonary…

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Added by PPMD on May 22, 2014 at 1:00pm — No Comments

SMT C1100 Preliminary Results from Phase 1B Clinical Trial for Treatment of Duchenne

In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profile in twelve boys with Duchenne. Plasma levels of the drug were variable and the company speculates that diet and the course of the disease may be the cause.



The preliminary trial data will be reviewed…

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Added by PPMD on May 21, 2014 at 9:00am — No Comments

Halo Files "Complete Response" with FDA regarding Clinical Hold of HT-100 for Duchenne

Since being placed on clinical hold in December, Halo has generated additional data from the HT-100 clinical program and completed several toxicology studies, and provided this information to…

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Added by PPMD on May 13, 2014 at 3:00pm — 1 Comment

Results of PPMD-Funded Study Prompt Phase III Study of Tadalafil in Duchenne

A recent PPMD-funded study at Cedars Sinai Medical Center in Los Angeles (published in the May 7, 2014, online issue of Neurology®) demonstrates that those with Duchenne have impaired circulation in their muscles, which may contribute to muscle weakness, and that the approved vasodilator tadalafil can restore that circulation to normal levels. …

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Added by PPMD on May 7, 2014 at 4:00pm — No Comments

Prosensa Update on Drisapersen: Re-dosing plans are underway & 96-week data from an open-label extension study presented at the American Academy of Neurology (AAN)

Prosensa Provides Update on Drisapersen

Re-dosing plans are underway and 96-week data from an open-label extension study of drisapersen for the treatment of DMD presented at the American Academy of Neurology (AAN)

Read the press release.…

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Added by PPMD on May 1, 2014 at 10:30am — 1 Comment

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