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Pfizer gene therapy for Duchenne fails to meet goals of key trial

BioPharma Drive: Drug Pricing

The negative results from the Phase 3 study, called Ciffreo, come a little more than a month after Pfizer said a patient died in another trial of the gene therapy.

Therapies 117
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Trial Confirms 'Life-Changing' Impact of Gene Therapy, Beqvez, for Hemophilia B

Drugs.com

25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows. THURSDAY, Sept. People with hemophilia B saw their bleeding episodes.

Therapies 116
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100x improvement in sight seen after gene therapy trial

Science Daily: Pharmacology News

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

Therapies 124
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New Gene therapy trial shows restored hearing and speech in children born deaf, treated in both ears

Science Daily: Pharmacology News

A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.

Therapies 118
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Trial Confirms 'Life-Changing' Impact of Gene Therapy for Hemophilia B

Drugs.com

25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows.People with hemophilia B saw their bleeding episodes. THURSDAY, Sept.

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How long do clinical trial phases take?

Antidote

Before a potential new therapy can be administered to patients, it must go through several clinical trial phases designed to test the drug or device for safety and effectiveness. For many new treatments, it will take approximately ten years for the therapy to progress from initial discovery to being approved.

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How long do clinical trials take?

Antidote

Before it is released onto the market, the development of any new drug or medical device must undergo rigorous testing , part of which involves clinical trials. Clinical trials are integral to making sure that any new therapy is both safe and effective for individuals, and volunteers are a vital part of the process.