SMA type 1

June 14, 2019June 14, 2019

Zolgensma ‘Saved All of Our Lives’: A Family’s Journey Through Gene Therapy

News

Tina and Torence Anderson’s sixth and youngest child, Malachi, came into the world on July 31, 2015. “When he was born, he was normal,” Tina said in a phone ... Read more

May 24, 2019May 26, 2019

FDA Approves Zolgensma, 1st Gene Therapy to Treat SMA in Children Up to Age 2

News

Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through ... Read more

May 8, 2019May 8, 2019

#AANAM – Zolgensma Continues to Extend Survival in SMA Type 1 Patients Several Years After Dosing

News

A single administration of Zolgensma continues to extend the survival of patients with spinal muscular atrophy (SMA) type 1 while halting motor neuron loss several years after dosing, according to long-term ... Read more

April 22, 2019May 6, 2019

Zolgensma Defying SMA Type 1 Natural History in STR1VE, Helping Treated Babies to Breathe and Sit, Early Data Show

News

Zolgensma (AVXS-101), an investigational gene therapy for patients with spinal muscular atrophy (SMA), improves motor function and prolongs survival in infants with SMA type 1 under six months of ... Read more

March 13, 2019March 28, 2019

Skin Lesions of Infant with SMA1 Cured After Spinraza Treatment, Case Study Reports

News

Treatment with Spinraza (nusinersen) was able to resolve many severe and widespread skin lesions in a 7-month-old infant with spinal muscular atrophy (SMA) type 1, a case report shows. ... Read more

December 4, 2018January 8, 2019

Zolgensma (AVXS-101) Under Priority FDA Review as Possible Gene Therapy for SMA Type 1

News

The U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that Zolgensma (onasemnogene abeparvovec-xxxx) — previously called AVXS-101 — be approved as a ... Read more

November 16, 2018November 20, 2018

LMI070 Oral Candidate For SMA Type 1 Holds Promise, Preclinical Study Shows

News

LMI070 (branaplam), a candidate oral therapy under development by Novartis, is being tested in a Phase 1/2 clinical trial for infants with spinal muscular atrophy (SMA) type 1. A recent study ... Read more

November 9, 2018November 14, 2018

Possible $4M Price for AVXS-101, ‘Foundational’ SMA Gene Therapy, Could Be Cost-Effective, Novartis Says

News

Novartis may put a $4 million to $5 million price tag on its “one-time, potentially curative” gene therapy AVXS-101 for spinal muscular atrophy (SMA) and believes this could be cost-effective. ... Read more

November 5, 2018November 16, 2018

Coordinators Are Key to Successful Family-centered Care for Kids with Type 1 SMA

News

Establishing care coordinators is key to providing successful family-centered care to young spinal muscular atrophy (SMA) type 1 patients and their families, according to a recent study. The study, “Identifying ... Read more

November 1, 2018November 5, 2018

Risdiplam Showing Potential to Raise SMN Protein to Healthy Levels in SMA Babies and Adults, PTC Says in Interview

News

Preliminary data from two ongoing clinical trials into risdiplam (RG7916) — a potential oral therapy for all types of spinal muscular atrophy — support its considerable promise, with treated babies ... Read more

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