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Gene therapy restores vision in first-of-its-kind trial

Drug Discovery World

A gene therapy developed by University of Florida scientists restored useful vision to most patients with rare condition Leber congenital amaurosis type I (LCA1) in a small trial. The trial also tested the safety profile of the treatment. Side effects were largely limited to minor surgical complications.

Therapies 173
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Trial to test monoclonal antibody for virus of growing concern

Drug Discovery World

The National Institutes of Health (NIH) is sponsoring a clinical trial to evaluate the safety of an investigational monoclonal antibody to treat enterovirus D68 (EV-D68), which can cause severe respiratory and neurological diseases such as acute flaccid myelitis (AFM). Credit: NIAID and CDC.

Virus 147
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Add-on treatment for severe uncontrolled asthma approvedĀ 

Drug Discovery World

Owing to the complexity of severe asthma, many patients have unclear or multiple drivers of airway inflammation resulting in some patients not responding to the current standard of treatment, and being at increased risk of asthma attacks, hospitalisations, and a poor quality of life 3,4,5,6. Final draft guidance is subject to appeal.

Treatment 246
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Bayer gets go-ahead for Huntingtonā€™s Disease gene therapy trialĀ Ā 

Drug Discovery World

A Phase I/II clinical trial targeting a treatment for Huntingtonā€™s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . In 2019, BV-101 was granted orphan drug designation in the European Union. . There are currently no approved disease modifying therapies for Huntingtonā€™s Disease.

Therapies 246
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Integration of AI in imaging clinical trialsĀ 

Drug Discovery World

Sophie Winandy , Executive Director of Medical Imaging at ICON explores the benefits of AI and how it can be integrated throughout medical imaging within clinical trials. This can lead to generation of more objective endpoints that might lead to a better assessment of treatment efficacy. The benefits of AI are many.

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Diabetic eye disease: The urgent need for early, non-invasive treatments

Drug Discovery World

Despite affecting such a large subset of diabetic patients, treatment options are limited to repeated, invasive intravitreal injections to the eye. Diabetic retinopathy ā€“ current treatments The journey for patients with DR is one of increasing severity and loss of vision. In 2019 there were >14.2 In 2019 there were >14.2

Disease 162
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ā€˜Ice Bucket Challengeā€™ funds FDA-approved ALS treatment

Drug Discovery World

The US Food and Drug Administration (FDA) has granted approval to AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS). million from the ALS Association, raised by the 2014 social media craze the ā€˜Ice Bucket Challenge’ Another 40 potential new treatments are being investigated. .