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 ...
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 ...
Fondazione Telethon ETS' Waskyra has become the first gene therapy in the US for patients with Wiskott-Aldrich syndrome (WAS) ...
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