The group knew that Samulski didn't have much business experience, Howell says."But, on the other hand, he knew a tremendous lot about viruses and how they work and how they might really be effectively brought into the clinical practice.""I remember Jude saying we probably won't get funding because gene therapy is an unproven technology and there are a lot of naysayers," saysToday, AskBio occupies a gleaming new headquarters in Research Triangle, N.C.
Then they started making deliveries — first in test tubes, then in mice, then in golden retrievers with a genetic mutation similar to the one Conner has. "They were able to run and jump," Jessica says."We saw this with our own eyes. And we just thought, 'Oh gosh, if one day Conner could get a chance to get something like this ...' — it just gave us so much hope."Playing with his brothers, Conner Curran says the gene therapy has really helped him."I can run faster. I stand better," Conner says.
To Conner, the treatment with what he calls"muscle juice" was no big deal."They put a needle in my arm for two hours," he says, when I ask him what it was like.The scientist was with the family in the days after Conner was infused with billions of viruses. He was also there days later when Conner became feverish and stopped eating — a common reaction to this type of treatment.
hassankhaderali Oh my God, this is amazing news!!
Great story NPR, now how about one on the truth about hydroxycloriquine saving lives but docs and pharma will not allow patients to have this inexpensive & effective medication.
Wow! Amazing what science is capable of. Keep up the life changing work ask_bio
The PHARMA is not happy with this; they'll lose billions... and their $$$ profits are priority of over peoples' lives! Shameful what this country has become! So embarrassed to be American!
Awesome 👍 A big applause to the brave parents who volunteer to be guinea pigs.
JoshuaGrubbsPhD Reminds me of our convo in St. Louis. Makes me wonder if my boys' genetic mutation is something that could be in this same universe of treatments for genetic diseases. The whole 'can't build muscle he's already lost' is big, tho.
So how much is Pfizer is going to charge once this therapy goes public?
junior_miller
Hope and pray it works
That's wonderful to hear!
finally ! actual news !
Now this is a big deal.
Encarnación Bail Romero who's son Carlitos was taken away from her was never news for you.
Jerry Lewis would be proud. Amazing stuff.
🙏🏾💜