XTalks

JUNE 8, 2021

In addition, recent data from a Phase I trial has shown ‘remarkable’ long-term results of the gene therapy in children with the disease. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein. The complex is important for the maintenance of motor neurons.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

pharmaphorum

JANUARY 9, 2023

SMA is a genetic disorder that can cause rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

Gene Therapy 119

Gene Therapy Genetics Gene Clinical Trials 119

pharmaphorum

AUGUST 10, 2020

Evrysdi is the third treatment approved for SMA , an ultra-rare muscle wasting disease that can begin in early childhood, after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma (onasemnogene abeparvovec). Evrysdi is designed to treat SMA by increasing production of the survival of the motor neuron (SMN) protein.

FDA Approval 59

FDA Approval Drugs Gene Therapy Protein 59