A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...
A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...
The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...
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Gene Therapy Maintains Efficacy in Hemophilia B Out to 5 Years
Based on initial positive results from HOPE-B, etranacogene dezaparvovec became the first gene therapy approved for adults ...
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...
Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike to ...
The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...
The patchwork rollout of a gene therapy exposes weaknesses in the pricing and regulatory frameworks that determine who ...
To our immune system, a potentially lifesaving gene therapy can look a lot like a dangerous infection. That's because most ...
The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
Hemophilia gene therapies arrived a few years ago with blockbuster expectations. But now, one therapy has been pulled from ...
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