On October 23, 2012, PPMD hosted a webinar with Sarepta to discuss of the 48 Week dystrophin and 6-minute walk test (6MWT) data from the Phase IIb extension study in Duchenne as well as next steps for the eteplirsen program. Chris Garabedian, President and CEO of Sarepta Therapeutics presented the data and answered submitted questions. Listed below are the outstanding questions addressed by Sarepta:
The safety profile of eteplirsen was evaluated across all patients through week 62 and there were no clinically significant treatment-related adverse events, no serious adverse events, and no discontinuations. One patient had a laboratory treatment-related adverse event, a transient elevation of urine protein on a urine dipstick test, however this elevation was not observed on a 24-hour urine protein measurement and resulted in no clinical symptoms or interruption of treatment. This patient did not show elevations of the specific renal markers of cystatin C or KIM-1. Across both the treatment and placebo/delayed treatment cohorts there is evidence of continued stabilization on pulmonary function tests, echocardiogram, muscle strength and clinical laboratory tests over the 62 weeks.
As patients and caregivers you have a powerful voice in educating about the disease and its impact. We would recommend that parent’s connect with existing organizations that are supportive of the interests of all patients, such as PPMD, who are undertaking important initiatives to efficiently and responsibly meet patient needs. We believe by pooling your support and resources behind these types of established initiatives you will be able to effect the most change.
We will keep the patient community informed as we make progress on the design and planning of any additional clinical studies through organizations such as PPMD. We will also have updates on our website as well as on clinicaltrials.gov that will include any clinical trial information and the contact information for clinical trial sites as they open for enrollment. If a patient or family would also like to receive Sarepta updates via email please send an email to firstname.lastname@example.org and ask to be included on our distribution list.
Sharon Hesterlee, Ph.D.
Vice President, Research
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