SMA type 1

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 5, 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

October 19, 2018October 24, 2018

AVXS-101 May Be ‘Transformative’ Breakthrough for SMA Type 1, Cure SMA President Says

News

AveXis‘ gene therapy AVXS-101, now under review for approval to treat infants with spinal muscular atrophy (SMA) type 1 in the U.S. and Europe, may be truly “transformative,” said Kenneth Hobby, president of ... Read more

October 3, 2018October 11, 2018

SMA Type 1 Shows Different Disease Course Between Plain Communities, Study Shows

News

Spinal muscular atrophy (SMA) type 1 has different disease courses between Plain (Amish and conservative Mennonite) communities, a recent natural history study shows. The study, “Spinal muscular atrophy within ... Read more

August 20, 2018September 10, 2018

Psychological Support Relieves Anxiety of Spinraza Treatment in Kids with SMA Type 1, Study Says

News

Psychological interventions to control emotional stress can help relieve the anxiety that parents and children with spinal muscular atrophy type 1 feel during Spinraza administration through spinal taps, an ... Read more

June 8, 2018November 9, 2018

AveXis, Maker of SMA Therapy AVXS-101, Expanding Manufacturing Operations to North Carolina

News

AveXis is expanding its operations to North Carolina, opening up a new manufacturing center in Durham County, North Carolina Gov. Ray Cooper recently announced in a press release. The company ... Read more

May 16, 2018July 6, 2018

Patients with SMA Types 2 and 3 at Risk of Malnutrition, Altered Metabolism, Study Reports

News

Patients with spinal muscular atrophy (SMA) types 2 and 3 are at risk of malnutrition, altered metabolic rates, and changes in body composition, a study reports. The study, “Comprehensive ... Read more

May 14, 2018July 6, 2018

Spinraza Improves Motor Skills in Broad Range of SMA Type 1 Children, EAP Study Shows

News

Spinraza (nusinersen) improves motor skills in a broad range of children with spinal muscular atrophy (SMA) type 1, including those in more advanced disease stages, according to a study of patients ... Read more

April 27, 2018April 27, 2018

First Patient Dosed in Phase 3 Trial of AVXS-101 in Presymptomatic Infants with SMA Types 1, 2 and 3

News

The first patient has been dosed in the Phase 3 clinical trial of AveXis’ gene therapy candidate AVXS-101 in presymptomatic infants with spinal muscular atrophy (SMA) types 1, 2 and 3, the company announced. ... Read more

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