July 2, 2014 / Comments Off
By Christine McSherry
Last month, members of the Duchenne Alliance including the Jett Foundation’s Christine McSherry and Jenn McNary; Tracy Seckler from Charley’s Fund and fellow patient advocate, Mindy Leffler had a private meeting with Senator Elizabeth Warren in her Cambridge, MA offices to discuss ways to keep the dialogue going about Duchenne. The Senator’s longtime health care advisor in Washington and avid supporter of our cause, Remy Brim PhD, also joined us that day.
Over the past several years, members of the Duchenne Alliance – parent advocates and supporters – have had the unique experience of dealing first-hand with the FDA as it works to implement the mandates set forth in the FDA Safety and Innovation Act (FDASIA), and as they relate to new treatments to stop the advancement of Duchenne Muscular Dystrophy. We believe our perspective may be useful to committee members as they consider how best to ensure the soonest possible access to safe and effective therapies for the treatment of the disease.
Our first objective for the meeting was to thank Senator Warren and Remy for their efforts to date in encouraging the FDA to follow FDASIA regarding Duchenne and the drug Eteplirsen from Sarepta Therapeutics. Shortly following Sarepta’s public announcement that they were given a clear pathway for drug approval, Prosensa, another pharmaceutical company in the Duchenne RNA therapeutic space, announced that they, too, received unprecedented news from the regulators at the FDA about their drug Drisapersen – demonstrating an extraordinary level of flexibility by the agency. During our visit, we also thanked the Senator for signing off on another Duchenne community initiative, the re-authorization of the MD – CARE Act, which has been extremely successful in creating additional programs in the Duchenne community since its inception in 2001 by Parent Project Muscular Dystrophy. We respectfully added, however, that there is still much work t
Gun shy from Lawyers ,do not expect anything unless it is in writing from who ever...haa.
he does seem weak on partnership but i think he wants all to be done by Sarepta ..
You cannot change a short term holders mind and you cannot make a bad researcher understand Sarepta's future.Only the true smart investors will buy and hold the rest will just jump on when ever news
The market place for a several safe exon skipping drugs such as Sarepta's could be worth as much as 20 billion in sales a year 'with in a few years ''unlike toxic other exon skipping drugs not good for long term dosing''
Sarepta has the market... simple facts
dosing every two days would create the build up much faster and then once weekly....]]the key is getting the buibuild up]]
The real fact is that it takes a while for pmo-s to build up in the body but once they do it works well.
WHEN EVER THE TRIAL STARTS COUNT 24 WEEKS AND THEN YOU WILL SEE A DRUG APPROVAL.THE LACK OF DYSTROPHIN PRODUCTION IS THE CAUSE OF DMD.NOW I AM ONE THAT WOULD SAY '' YOU COULD START DOSING AT 50M UNTIL PRODUCTION IS THERE AND THEN CUT BACK TO 30 AT 20 WEEKS'' YOU MAY GET THERE A BIT QUICKER.
Eteplirsen Led to Dystrophin Production in US DMD Trial
Twenty-four weeks of treatment with the exon-skipping drug eteplirsen resulted in significant increases in dystrophin production
IT takes a while to build up pmo-s into the body ,so one could believe the more loaded into body the quicker production but once production starts from that buildup you need only 30m per week..