Sarepta Rare Disease Advisory Panel Meeting today, page-11

More on DMD trial news by Summit Therapeutics for FDA UK/US extension which does not limit dystrophin gene limitation as Sarepta and others do:

06:46 26 Apr 2016
FDA clears Summit Therapeutics to extend
PhaseOut DMD trial to the US

Shares in Summit Therapeutics PLC (LON:SUMM, NASDAQ:SMMT) perked
up after the drug developer revealed it had received some good news from US
regulators.
The US Food and Drugs Administration (FDA) has cleared the company's
investigational new drug (IND) application to expand the Phase II proof-ofconcept
clinical trial, known as 'PhaseOut DMD', in the US.
PhaseOut DMD will evaluate the company's lead utrophin modulator, ezutromid
(formerly known as SMT C1100), in patients with Duchenne's muscular
dystrophy (DMD), at sites in the UK and the US.
In contrast to many current therapeutic approaches to DMD, utrophin
modulation has the potential to treat all boys and young men with DMD,
regardless of their underlying dystrophin gene mutation, Summit's statement
said.
DMD is a progressive muscle wasting disease that affects around 50,000 boys
and young men in the developed world, generally leading to death by the late
twenties. The disease is caused by different genetic faults in the gene that
encodes dystrophin, a protein that is essential for the healthy function of all
muscles.
"The IND clearance for PhaseOut DMD paves the way to expand PhaseOut
DMD into the US and will provide access to a wider network of leading
physicians in DMD as we seek to improve the lives of patients and families
living with this devastating disease," said Ralf Rosskamp, MD, chief medical
officer of Summit.
Summit expects to begin enrolling patients in the US in the third quarter of this
year.
"PhaseOut DMD aims to show the potential benefits of ezutromid as a disease
modifying approach for DMD for the first time in patients, and this approach
could ultimately benefit the entire DMD patient population," Dr Rosskamp said.​

Shares in Summit were up 4.8% at 111.65p in lunchtime trading.