A clear collection of evidence that this drug is changing the natural history of the DMD disease .
the best investments are biotechs for 2014 .........................................
In the study, PPMO was injected into mice models with two strains of Acinetobacte that often cause infection and affects hospitalized patients with weakened immune systems, chronic lung disease or diabetes. Acinetobacte is considered as one of the top bacterial infection threats in the U.S. due to its resistance against many antibiotics.
PPMO succeeded in reducing the number of infectious bacteria in mice by more than 90%. Survival rate of infected mice was also improved by PPMO treatment.
”We set out to target specific genes in Acinetobacter in an effort to inhibit the bacterium’s growth,” said Dr. David Greenberg, assistant professor of internal medicine and microbiology and senior author of the study. The technology that created the synthetic PPMO could be used to develop similar antibiotics targeting other bacteria and viruses, he added. ”We believe there is a lot of promise in developing new antibiotics that target specific pathogens as opposed to so-called broad-spectrum antibiotics that target whole classes of bacteria.”
The FDA needs classes on these types of drugs so they can get a better understanding and the big pharma needs to get off the old ''off target medicines'' and move to a direct or more exact drug platforms...wake up to the future boys.
yes i agree that a small safety/ production of each exon for maybe 26 weeks would be ok.Again we are still talking about a targeted exon with the same drug of a different sequence that is made exactly for it's target.Facts are that it will work when directed correctly with the drug target it is made for..no way around that...............
2012 was a good approval year 2013 was a poor year ....take notice that 2014 may be the record breaker..
A phase I/IIa clinical trial in Duchenne muscular dystrophy using systemically delivered morpholino antisense oligomer to skip exon 53
Duchenne muscular dystrophy (DMD) is a progressive, lethal muscle degenerative condition arising from the absence of dystrophin in skeletal and cardiac muscles. 65% of DMD boys have out-of-frame deletions. Modulation of pre-mRNA splicing by exon skipping is the most promising molecular intervention in DMD. 2 Phase Ib and 2 Phase IIa clinical trials (MDEX Consortium in collaboration with Sarepta Therapeutics; a Dutch Consortium) demonstrated that delivery of antisense oligonucleotides (AOs) to mediate exon skipping of exon 51 were able to return specific DMD mutations in-frame (~13% of all mutations) leading to new dystrophin protein expression after intramuscular and systemic delivery. The Dutch study of repeated 2-O-methylated phosphorothioate (2OMe) AO administration suggested limited efficacy after 5 weeks of treatment. Our MDEX Consortium study using a morpholino (PMO) AO demonstrated a clear dose response, robust dystrophin restoration and reduction of muscle inflammation after 12 weeks at doses up to 20mg/Kg, with no drug related adverse events. This, and preclinical studies focused on level of protein expression, clearly indicate that PMO have a superior therapeutic index compared to 2OMe.
New PMOs are needed to target other DMD mutations. We will develop a PMO to skip exon 53 and perform a clinical trial in DMD boys using a world leading pan-European consortium. This will allow us to advance this class of PMO therapy in DMD by:
i. Assessing the safety and efficacy of targeting another exon;
ii. Exploring the use of non-invasive techniques to monitor dystrophin restoration.
This new PMO will be administered over 12 weeks in 3 groups, each of 4 DMD boys, receiving between 4 to 30mg/kg or placebo
March 5, 2014
06:09 EDT SRPT, RNA Prosensa shares could be worth $29 with early filing, says Citigroup
Citigroup raised its price target for shares of Prosensa (RNA) to $7.50 from $4 but says the stock could be worth $29 with an early filing for its Duchenne muscular dystrophy treatment. Citi says that in comparison to Sarepta (SRPT), a company with a competing DMD treatment, Prosensa’s stock is cheaper, but notes that it believes Sarepta's eteplirsen is more potent in terms of dystrophin production in animal models. Citi keeps a Neutral rating on Prosensa.