Charlie And Rupert On Their Journey With Spinal Muscular Atrophy And Zolgensma Gene Therapy

Charlie and Rupert on their journey with Spinal Muscular Atrophy and Zolgensma Gene Therapy

Wonder drug gives Izzy chance to be ‘normal, healthy child’

Spinal Muscular Atrophy (SMA), Zolgensma, Gene therapy approved by NHS, #SMA, #nhs , #zolgensma

Spinal Muscular Atrophy gene therapy - gene therapy for spinal muscular atrophy

HPR

Two Siblings with SMA Receive Gene Therapy

Understanding ZOLGENSMA

MDA

Dr. Shephard Mpofu, Novartis, Discusses Gene Therapy for Children with Spinal Muscular Atrophy

Riley's story: One of the first babies in the South West to receive Zolgensma gene therapy treatment

University Hospitals Bristol and Weston NHS FT

Family turning to million-dollar drug for son

Baby becomes first NHS patient to get drug for genetic condition

The Independent

Long-Term Data for SMA Gene Therapy Continues to Show Efficacy

Understanding Spinal Muscular Atrophy (SMA)

SMA Type 2: A Diagnostic Journey

New Therapies are Changing the Outcomes of SMA

The France Foundation

April 2022 Cure SMA and Novartis Gene Therapies Clinical Trial Update Webinar

Two toddlers win worldwide drug lottery

Hamilton Health Sciences

Ruzgar's story with Spinal Muscular Atrophy (SMA)

CHRISTUS Health

Life Saving Spinal Muscular Atrophy (SMA) Treatment | Sam's Story

Johns Hopkins Medicine

SMA Type 1: How Gene Therapy Works

NationwideChildrens

Lunch & Learn: Gene Therapy for SMA