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UCLA Gene Therapy Achieves 100% Cure Rate for Bubble Baby Disease in Clinical Trials

11 years ago

UCLA researchers led by Dr. Donald Kohn have successfully cured all 18 children with ADA-deficient SCID using a novel stem cell gene therapy approach over two clinical trials since 2009.
The breakthrough treatment uses patients' own genetically modified blood stem cells to restore immune function, eliminating the need for lifelong enzyme injections or risky bone marrow transplants.
All treated patients, ranging from 3 months to 4 years old, developed fully functioning immune systems without side effects and can now live normal lives.
The team is seeking FDA approval for the therapy and plans to extend the approach to treat sickle cell disease in clinical trials beginning in 2015.

13 years ago

Cook Medical's Zilver PTX drug-eluting peripheral stent achieved FDA approval as the first device of its kind approved for peripheral artery use in the United States.
The device combines mechanical stenting with paclitaxel drug coating to reduce follow-up procedures by more than 50 percent in femoropopliteal artery treatment.
OhioHealth Riverside Methodist Hospital became the first U.S. institution to commercially implant the Zilver PTX stent following FDA approval.
The stent maintains arterial blood flow in seven out of ten patients through 24 months after implantation in the superficial femoral artery.