Look what they did last year on biotech around the same time.they smashed a lot of small caps.
big monies and the government with tax collections.
Sarepta has all and much more than any other drug.
The new guidance takes into consideration increased risk tolerance among some DMD patients involved in trials. Acceptable endpoints may now include functional, cardiac, or respiratory metrics, though the FDA has asserted that the ideal trial design still involves a randomized, placebo-controlled design—even if the placebo-controlled part involves using historical data.
Hope thats the case all the time... ''AA''
also validates many billions in future sales...many
COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
Application number: 20150080311
Abstract: An antisense compound for use in treating myotonic dystrophy DM1 or DM2, a method of enhancing antisense targeting to heart and quadricep muscles, and a method for treating DM1 or DM2 in a mammalian subject are disclosed. The oligonucleotide has 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3?UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively. Conjugated to the oligonucleotide is a cell-penetrating peptide having the sequence (RXRR(B/X)R)2XB, where R is arginine; B is ?-alanine; and each X is —C(O)—(CH2)n—NH—, where n is 4-6. The antisense compound is effective to selectively block the sequestration of muscleblind-like 1 protein (MBNL1) and/or CUGBP, in heart and quadricep muscle in a myotonic dystrophy animal model.
Filed: April 24, 2014
Issued: March 19, 2015
Assignee: Sarepta Therapeutics, Inc.
Inventors: Hong M. Moulton, Ryszard Kole
Roth Capital reaffirmed their buy rating on shares of Sarepta Therapeutics, Inc. (NASDAQ:SRPT) in a research note issued to investors on Monday. Sarepta Therapeutics, Inc. (NASDAQ:SRPT) belongs to Healthcare sector. Its weekly performance is 5.31%. On last trading day company shares ended up $33.70. Sarepta Therapeutics, Inc. (NASDAQ:SRPT) distance from 50-day simple moving average (SMA50) is 26.68%.