Hearing these stories kills me just like the PCH is keeping PBT2...

Hearing these stories kills me just like the PCH is keeping PBT2 from HD and AD sufferers-
Sarepta's Duchenne drug trial hits 5-year mark: Parents look back

Ana Vaish considers Aug. 15 to be the date of “a life-changing event” for her and her family.
Five years ago on that day, Vaish was in a hospital room at Nationwide Children’s Hospital in Ohio watching her 9-year-old son, Ryan, being infused. It was the first administration in a trial of a drug, called eteplirsen, that might help slow his disease, Duchenne muscular dystrophy. The Los Angeles-area resident had been accepted into the placebo-controlled, 12-patient trial of the drug developed by a Seattle-based biotech then called AVI BioPharma, which later moved to Cambridge and was renamed Sarepta Therapeutics (Nasdaq: SRPT). She and her son had taken an early-morning flight to Columbus, Ohio the day before in order to receive the infusion. They would make that same weekly trip for the better part of a year.
Ryan’s health had been declining rapidly in the months before the trial started, said Ana Vaish in an interview last week. “We had noticed things were getting harder for him and he was making compensations for things,” she said.
No one knew at the time whether Ryan was being infused with the actual drug or with a placebo. But within a couple of months, Ana Vaish started to have suspicions. She remembers watching her son come home from the fourth grade and jump into the pool rather than sit and rest.
“It was not right away. But in a couple months, I would see a real boost in energy for him,” she said. “I would be like, ‘There’s something going on.’... in the back of my head, I said, ‘I think he’s on the medication.'”
Ana Vaish learned in the spring of 2012 that Ryan had indeed been in the arm of patients who received eteplirsen. He has continued to receive the drug once a week, as the company that developed the drug extended what was initially a six-month, placebo-controlled trial into an open-label one.
All 12 of the boys in the trial — who range in age from 12 to 15 — are now receiving the actual drug, and 10 of them are still walking, according to interviews with several of the families last week. The average age at which Duchenne forces patients into wheelchairs is 12.
The drug is now under review by the Food and Drug Administration for accelerated approval, and it’s an understatement to say the decision is beingwatched worldwide. No FDA decision regarding a drug this year will have more far-ranging effects, both for thousands of families of boys with Duchenne and for the entire field of rare drug development. But the decision has been delayed for months now, as some officials at the FDA have argued that the trial is too small to prove the drug is effective, even on a temporary basis until the results of a larger, confirmatory trial. No one knows when the agency might announce its decision.

More:
http://www.bizjournals.com/boston/b...enne-drug-trial-hits-5-year-mark.html?ana=twt