stock moving a bit
Nov. 18, 2014 | 12:01 am
Christine McSherry's son Jett is a typical college freshman trying to push for as much independence as he can. But Duchenne muscular dystrophy, a disease that tears away muscles, has taken his ability to walk. He relies daily on aides to help him with homework, eating and carrying classroom materials.
"He feels he has regressed to being a young child," McSherry says. "It's a horrible paradigm to be in."
Jett is planning on majoring in history and is enjoying the social aspects of his college outside of Boston. Soon, he'll begin a clinical trial that he's been waiting two years to participate in, and his family hopes the experimental drug – eteplirsen, made by Sarepta Therapeutics – will stabilize his condition and give him a longer life.
There is no cure for Jett's condition, which is fatal, but parents like McSherry believe there is hope if they can bypass government bureaucracy and gain access to medications still being tested by scientists. "If there was a drug to treat my son's disease, then he would at least have the right to try it without long and expensive waits for it," she says.
Some states are making this process easier, passing laws that allow terminally ill patients access to drugs that have not been approved by the government. In Arizona, such a provision quietly won approval during the midterm elections.
A map depicting which states have "right to try" statutes.
This type of measure – passed also in Colorado, Louisiana, Michigan and Missouri – is known as a "right to try" statute. It allows dying patients to request access to treatment that has not been approved by the Food and Drug Administration, the federal agency that reviews testing by drug companies to decide whether medications are safe and effective. The laws allow doctors, hospitals and manufacturers to bypass the FDA and protect them from
It would be nice if they had new news for us
very happy but keep in mind how many children are not being allowed to take this drug. they are quickly deteriorating to a point where if/when the fda finally approves, it may be too late. imagine if we started treatment early on. perhaps the progression would be so slow that these kids could live somewhat normal lives. but hell, the fda is compassionate. they approved poison like zohydro and approved 3 obesity drugs with bad side effects. way to go fda!!!! who cares about sick, dying kids when we can fight off obesity!
I know there is no one here
Everyone is a tough guy when they can something without being found out.
jenn mcnary @jennmcnary 13m13 minutes ago
$SRPT dosing patient number 14- our dear friend Aiden - happy doesn't begin to describe the feeling
Isn't it "you're" embarrassing "yourself"? It seems that "you're" now "embarrassing" yourself.
upgrades flowing in
Sarepta Therapeutics, Inc. (SRPT), a developer of RNA-based therapeutics, today announced that it has initiated dosing in a clinical study of eteplirsen, the Company's lead exon-skipping therapeutic candidate for the treatment of Duchenne muscular dystrophy (DMD), in patients who are non-ambulant or who have advanced DMD and don't meet a minimum 6-minute walk test score at baseline.
The open-label study, 4658-204 (Study 204), will include approximately 20 patients treated with eteplirsen who have genotypes amenable to exon 51 skipping and who meet other study inclusion criteria. The study will be conducted at several sites in the United States and is designed to evaluate the safety of eteplirsen in DMD patients over 96 weeks of dosing. Patients enrolled in the study will receive once weekly intravenous infusions of 30mg/kg of eteplirsen, and data will be collected across a number of safety parameters and secondary efficacy endpoints.
“The initiation of this eteplirsen study represents an important milestone for patients, their families, and the DMD community,” said Edward Kaye, M.D., Sarepta’s Chief Medical Officer. “Expanding the DMD population to include patients who are older and non-ambulant demonstrates our strong commitment to develop eteplirsen for patients at all stages of DMD and will provide additional data to support our planned NDA filing.”
Fawn Leigh, M.D., of Mass General Hospital and a principal investigator in the study added, "Eteplirsen is a potential breakthrough treatment for patients with DMD. I am pleased to be able to offer this promising disease-modifying treatment to my patients and to potentially alter the course of this devastating disease.”
The stock needs something to stop the bleeding
Excellent summary immediate. After hearing the presentation and reading the comments from the parents on twitter, it is nothing short of a crime that the FDA has continued to delay this drug. The FDA should pay for this. I guess approving poison like zohydro and 3 obesity drugs is more important to them
Really scary FDA can get away with this
I'm confident too