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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 315
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Single-dose gene therapy is potentially life-changing for adults with hemophilia B

Science Daily: Pharmacology News

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.

Therapies 305
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Ventyx’s TYK2 drug suffers second setback

BioPharma Drive: Drug Pricing

The company claimed “higher than anticipated” scores from trial volunteers on placebo led to the disappointing data, prompting it to halt internal trials.

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Early-stage stem cell therapy trial shows promise for treating progressive MS

Science Daily: Pharmacology News

An international team has shown that the injection of a type of stem cell into the brains of patients living with progressive multiple sclerosis (MS) is safe, well tolerated and has a long-lasting effect that appears to protect the brain from further damage.

Therapies 242
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The Power of Worldwide Networks in Data and Safety Monitoring Boards

Advarra

In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Data and safety monitoring boards (DSMBs), also known as data monitoring committees (DMCs), play a critical role in overseeing a clinical trial’s safety and efficacy. local standards of care).

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How the FSP Model Augments Biotech Sponsors’ Internal Capabilities

PPD

FSP solutions are widely used in biotech Biotech companies of any size can benefit from using a contract research organization (CRO) to fulfill trial-related functions and services. With FSO, all tasks for a clinical trial are outsourced. are outsourced.

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Fitusiran

New Drug Approvals

2] History The efficacy and safety of fitusiran were assessed in two multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male participants with either hemophilia A or hemophilia B. [2] 2] [3] Names Fitusiran is the international nonproprietary name. [4] Fitusiran 1711.0g/mol, Fitusiran 1711.0g/mol,