SMA type 1

June 8, 2018June 8, 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

April 25, 2018June 18, 2018

#AAN2018 — RG7916 Works to Raise SMN Protein Levels in Babies with SMA Type 1, Trial Shows

News

A potential oral treatment for children with type 1 spinal muscular atrophy (SMA) called RG7916 was able to safely raise levels of the crucial SMN protein that is lacking in these patients, ... Read more

April 24, 2018April 26, 2018

#AAN2018 — Type 1 SMA Babies Treated with AVXS-101 Achieving New Milestones, Trials Show

News

Type 1 spinal muscular atrophy (SMA) babies treated with AveXis’ gene therapy candidate AVXS-101 continue to achieve unprecedented motor milestones and do not require breathing or nutritional support, according ... Read more

April 18, 2018April 27, 2018

#AAN2018 – SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show

News

Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) patients, according to soon-to-be-presented data from a Phase 2/3 clinical trial. The company will ... Read more

April 8, 2018July 6, 2018

SMA Expert Reveals Insights into Patient Care, Hope for Future Cure in Interview

News

Spinal muscular atrophy (SMA) expert Basil T. Darras, MD, is optimistic about treatment advances and the possibility of a future cure, according to a recent interview in which he shared ... Read more

March 23, 2018March 23, 2018

AveXis Plans a May Start for Phase 3 European Trial of Its SMA Type 1 Gene Therapy

News

AveXis’ expects to start a Phase 3 clinical trial of its spinal muscular atrophy (SMA) gene therapy AVXS-101 in Europe in May. The STR1VE-EU trial (NCT03461289) is expected to include up to ... Read more

February 13, 2018February 15, 2018

AVXS-101 Sets Out to Show It Might Be ‘Transformative’ for All SMA Types: Interview with AveXis

News

A broad range of clinical studies into AveXis’s gene therapy AVXS-101 is likely to decide whether it is indeed a “transformative” treatment for babies and children with spinal muscular atrophy (SMA) types ... Read more

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