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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

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Grand Rounds December 15, 2023: Diversifying Clinical Trials: A Path Forward (Roxana Mehran, MD, FACC, FAHA, MSCAI, FESC)

Rethinking Clinical Trials

years for females at age 45 years or older. Of those who have had a first MI, the percentage with a recurrent MI or fatal CHD within 5 years is 17% of males and 21% of females age 45 years or older. With the current ability to become pregnant this can affect women up to the age of 50. years for males and 5.5

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Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

XTalks

Elevidys was granted FDA accelerated approval for the treatment of pediatric patients four to five years of age with DMD who have a confirmed mutation in the DMD gene. Sarepta said it will continue evaluating Elevidys’ effectiveness among these ages in a confirmatory trial in hopes to remove age restrictions with an expanded label.

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Cannabinoids receptors: popular preclinical target but banned in 137 countries

Pharmaceutical Technology

Collectively, cannabinoid receptors (CB1 and CB2) are currently the most popular targets in preclinical stage of development, with 391 drugs tagged in total. This is closely followed by CB2 receptors in second place. This is followed by epilepsy at 8% of the pipeline and multiple sclerosis at 7% of the pipeline.

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Grand Rounds September 22, 2023: Integrating Community Health Workers into Team-Based, Early Childhood Preventative Care (Tumaini Rucker Coker, MD, MBA)

Rethinking Clinical Trials

The average child age was 4 months. Tags #pctGR, @Collaboratory1 The post Grand Rounds September 22, 2023: Integrating Community Health Workers into Team-Based, Early Childhood Preventative Care (Tumaini Rucker Coker, MD, MBA) appeared first on Rethinking Clinical Trials.

Trials 269
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Grand Rounds February 3, 2023: Pragmatic Trials For Children With Congenital Heart Disease – Insights From The NITRIC Trial (Luregn Schlapbach, PhD, FCICM)

Rethinking Clinical Trials

All infants and children less than 2 years of age undergoing open heart surgery on cardiopulmonary bypass were eligible for the study with exclusions for diseases that implied high severity pre-bypass. One of the main challenges of cardiac surgery, in addition to the technical complexities, is the occurrence of low cardiac output syndrome.

Trials 130
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Grand Rounds December 8, 2023: A Pragmatic Randomized Trial of the Jumpstart Intervention to Promote Communication about Goals of Care for Hospitalized Patients with Serious Illness (Ruth Engelberg, PhD; Erin Kross, MD; Robert Lee, MD, MS)

Rethinking Clinical Trials

Patients who were eligible had been hospitalized at least 12 hours but no more than 96 hours, age 55 and older with at least one Dartmouth Atlas chronic condition, or they were age 80 or older. It is something we are thinking about a lot in the outpatient trial. Clinicians could receive prompts on an almost daily basis.

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