REVIEW
Add like
Add dislike
Add to saved papers

Drug treatment for spinal muscular atrophy type I.

BACKGROUND: Spinal muscular atrophy (SMA) is caused by degeneration of anterior horn cells, which leads to progressive muscle weakness. Children with SMA type I will never be able to sit without support and usually die by the age of two years. There are no known efficacious drug treatments that influence the disease course.

OBJECTIVES: To evaluate if drug treatment is able to slow or arrest the disease progression of SMA type I, and to assess if such therapy can be given safely. Drug treatment for SMA type II and III will be will be the topic of a separate Cochrane review.

SEARCH STRATEGY: We searched the Cochrane Neuromuscular Disease Group Trials Register (September 30 2008, The Cochrane Library (Issue 3, 2008), MEDLINE (January 1966 to June 2008), EMBASE (January 1980 to June 2008), ISI (January 1988 to June 2008), and ACP Journal Club (January 1991 to June 2008).

SELECTION CRITERIA: All randomized or quasi-randomized trials that examined the efficacy of drug treatment for SMA type 1 were sought. Participants had to fulfil clinical criteria and, in studies including genetic analysis to confirm the diagnosis, have a deletion or mutation of the SMN1 gene (5q11.2-13.2)The primary outcome measure was to be time from birth until death or full time ventilation. Secondary outcome measures were to be development of rolling, sitting or standing within one year after the onset of treatment, and adverse events attributable to treatment during the trial period.

DATA COLLECTION AND ANALYSIS: Two authors (WB and AV) independently reviewed and extracted data from all potentially relevant trials. For included studies pooled relative risks and pooled weighted standardized mean differences were to be calculated to assess treatment efficacy

MAIN RESULTS: One small randomized-controlled study comparing riluzole treatment to placebo for SMA type 1 was identified and included in the review. Regarding the primary outcome measure three of seven children treated with riluzole were still alive at the age of 30, 48 and 64 months, whereas all three children in the placebo group died, but the difference was not statistically significant. Regarding the secondary outcome measures none of the patients in the riluzole or placebo group developed the ability to roll, sit or stand, and no adverse effects were observed.

AUTHORS' CONCLUSIONS: No drug treatment for SMA type I has been proven to have significant efficacy.

Full text links

For the best experience, use the Read mobile app

Group 7SearchHeart failure treatmentPapersTopicsCollectionsEffects of Sodium-Glucose Cotransporter 2 Inhibitors for the Treatment of Patients With Heart Failure Importance: Only 1 class of glucose-lowering agents-sodium-glucose cotransporter 2 (SGLT2) inhibitors-has been reported to decrease the risk of cardiovascular events primarily by reducingSeptember 1, 2017: JAMA CardiologyAssociations of albuminuria in patients with chronic heart failure: findings in the ALiskiren Observation of heart Failure Treatment study.CONCLUSIONS: Increased UACR is common in patients with heart failure, including non-diabetics. Urinary albumin creatininineJul, 2011: European Journal of Heart FailureRandomized Controlled TrialEffects of Liraglutide on Clinical Stability Among Patients With Advanced Heart Failure and Reduced Ejection Fraction: A Randomized Clinical Trial.Review

Get seemless 1-tap access through your institution/university

For the best experience, use the Read mobile app

Read by QxMD is copyright © 2021 QxMD Software Inc. All rights reserved. By using this service, you agree to our terms of use and privacy policy.

Get seemless 1-tap access through your institution/university

For the best experience, use the Read mobile app