The full text of this article can be found here:  https://link.springer.com/article/10.1007/s40265-018-0868-

A Aartsma-Rus

A Nolle

AK Bevan

AK Thomson

C Fallini

C Gouarne

C Sunyach

CA Chiriboga

CA Wijngaarde

CE Holt

CF Valori

CL Lorson

DT Hwee

E Bertini

E Dominguez

E Mercuri

E Somers

E Szunyogova

Ewout J. N. Groen

G Hamilton

G Sanchez

GE Oprea

H Zhou

Hannah K. Shorrock

IA Darbar

IEC Verhaart

JL Taylor

JR Mendell

JT Kissel

KA Kaifer

KD Foust

KJ Swoboda

KT Luu

L Garbes

L Pellizzoni

M Bowerman

M Feldkotter

M Riessland

M Scoto

MA Farrar

MA Lorson

MA Passini

MB Abera

MO Deguise

MR Lunn

N Miller

N Piazzon

NA Naryshkin

P Bernabo

PG Donlin-Asp

PJ Boyd

Q Liu

RA Powis

RI Wadman

RS Finkel

S Hosseinibarkooie

S Kariya

S Lefebvre

S Tisdale

SF Dobrowolski

SM Hammond

SM Hoy

T Bordet

T Shigeoka

TH Gillingwater

Thomas H. Gillingwater

TM Wishart

U Fischer

UR Monani

V Mariot

Y Hua

Z Zhang

English

Spinal muscular atrophy (SMA) is a neurodegenerative disease primarily characterized by a loss of spinal motor neurons, leading to progressive paralysis and premature death in the most severe cases. SMA is caused by homozygous deletion of the survival motor neuron 1 (SMN1) gene, leading to low levels of SMN protein. However, a second SMN gene (SMN2) exists, which can be therapeutically targeted to increase SMN levels. This has recently led to the first disease-modifying therapy for SMA gaining formal approval from the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). Spinraza (nusinersen) is a modified antisense oligonucleotide that targets the splicing of SMN2, leading to increased SMN protein levels, capable of improving clinical phenotypes in many patients. In addition to Spinraza, several other therapeutic approaches are currently in various stages of clinical development. These include SMN-dependent small molecule and gene therapy approaches along with SMN-independent strategies, such as general neuroprotective factors and muscle strength-enhancing compounds. For each therapy, we provide detailed information on clinical trial design and pharmacological/safety data where available. Previous clinical studies are also discussed to provide context on SMA clinical trial development and the insights these provided for the design of current studies.</p

Shorrock, Hannah K

Gillingwater, Thomas H

Groen, Ewout J N

Edinburgh Research Explorer

Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy

Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy

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