Rethinking Clinical Trials

SEPTEMBER 28, 2023

This may be the only interaction the family has with a healthcare professional, so if there are needs around behavior, health, social needs they will be addressed during these visits. Based on these findings, the research team initiated a larger PARENT trial that had 3 parent coaches at federally qualified health centers.

Trials 278

Trials Clinical Trials Clinical Trials Research 278

XTalks

JUNE 8, 2021

With a price tag of over $2.5 In England, five-month old baby Arthur became the country’s first SMA patient to receive the Zolgensma gene therapy last week through the National Health Service (NHS). They also advised use of Zolgensma for infants with SMA type 1 before they develop symptoms. Baby Arthur: First UK Patient.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

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 58

FDA Approval Drugs Gene Therapy Protein 58