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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

SEPTEMBER 28, 2023

The Parent-Focused Redesign for Encounters, Newborns to Toddlers (PARENT) study was a randomized controlled trial of PARENT verses usual care for parents with infants 12 months and younger over a 12-month study period. Adapted for early childhood preventive care, the structure should be team- and community-based.

Trials

Trials Clinical Trials Clinical Trials Research

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

FEBRUARY 13, 2023

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. . – Why didn’t you start to design the follow-on trial while you were still in the field?

Trials

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Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

XTalks

JUNE 8, 2021

With a price tag of over $2.5 They also advised use of Zolgensma for infants with SMA type 1 before they develop symptoms. Participants included symptomatic infants with SMA type I who had two copies of SMN2 who received prior treatment with low IV doses of Zolgensma in the trial. Why is Zolgensma So Expensive?

Gene Therapy

Gene Therapy Gene Protein Trials

Calamine lotion for chickenpox skin itchiness

Druggist

OCTOBER 25, 2020

Despite its higher price tag as compared with other products sold for the management of chickenpox PoxClin Cool Mousse is regarded as ‘highly-rated’ and well-priced product on Amazon.co.uk E45 Itch Relief Cream can be used in children and infants over the age of one month. Buy on Amazon Price incl. Buy on Amazon Price incl.

Production

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Roche takes on pricey rivals as FDA approves SMA drug

pharmaphorum

AUGUST 10, 2020

While this is not cheap by anybody’s reckoning, Roche hopes that the less intimidating price tag, plus the patient-friendly oral administration method will give it a competitive edge. Infants achieved the ability to sit without support for at least five seconds, a key motor milestone not normally seen in the natural course of the disease.

FDA Approval