Baby gene therapy study offers hope for fatal muscle disease – ABC News
Newsweek |
Baby gene therapy study offers hope for fatal muscle disease
ABC News A first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe has extended the tots’ lives, and some began to roll over, sit and stand on their own, researchers reported Wednesday. Only 15 babies with … Gene Therapy, New Drug Battle a Rare But Deadly Disease in KidsU.S. News & World Report The Most Expensive Drug in the World Is About to Be Made Worthless by Gene TherapyNewsweek New gene-replacement therapy shows promise for kids with neuromuscular diseaseThe Columbus Dispatch SMA News Today –Fox Business –Genetic Engineering & Biotechnology News all 8 news articles » |
Endpoints News |
AveXis offers compelling reasons for a fast FDA OK for gene therapy to treat spinal muscular atrophy in infants
Endpoints News But in a small Phase I study of 15 patients treated with AveXis’ $AVXS gene therapy for SMA, all of the patients were able to reach 20 months without the need for constant respiratory support, or dying. And many achieved improvements in motor symptoms, … |
U.S. News & World Report |
Baby Gene Therapy Study Offers Hope for Fatal Muscle Disease
U.S. News & World Report In this image provided by Nationwide Children’s Hospital, Dr. Jerry Mendell of the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio. Mendell led a small study of gene therapy in babies born with a usually fatal neuromuscular … Gene Therapy, New Drug Battle a Rare But Deadly Disease in KidsU.S. News & World Report The Most Expensive Drug in the World Is About to Be Made Worthless by Gene TherapyNewsweek Phase 1 study shows encouraging data for gene replacement therapy for SMA type IMedical Xpress The Columbus Dispatch –Fox Business –Genetic Engineering & Biotechnology News all 13 news articles » |
Science Magazine |
Gene therapy’s new hope: A neuron-targeting virus is saving infant lives
Science Magazine The results of the trial Evelyn participated in have blown away gene therapy researchers, too, marking one of the once-troubled field’s most dramatic successes yet. All 15 babies treated for SMA1, expected to die by age 2, are alive at 20 months or … |
Science Magazine |
Gene therapy’s new hope: A neuron-targeting virus is saving infant lives
Science Magazine The results of the trial Evelyn participated in have blown away gene therapy researchers, too, marking one of the once-troubled field’s most dramatic successes yet. All 15 babies treated for SMA1, expected to die by age 2, are alive at 20 months or … |
Science Magazine |
Gene therapy’s new hope: A neuron-targeting virus is saving infant lives
Science Magazine The results of the trial Evelyn participated in have blown away gene therapy researchers, too, marking one of the once-troubled field’s most dramatic successes yet. All 15 babies treated for SMA1, expected to die by age 2, are alive at 20 months or … |