Pharmafile Logo
Pharmafocus logo
Advanced Search

spinal muscular atrophy

Showing 15 posts of 20 posts found.

chuttersnap-oijvdm3zx4i-unsplash

Novartis shares new data for Zolgensma in children with SMA

March 5, 2024
Musculo-skeletal disorder, Novartis, Zolgensma, sma, spinal muscular atrophy

Novartis has announced new data to continue the support of the clinical benefits of Zolgensma (onasmenogene abeparvovec), for the treatment …

News

NMD Pharma begins phase 2 trial for spinal muscular atrophy treatment

September 27, 2023
Research and Development Musculo-skeletal disorder, NMD Pharma, clinical trial, spinal muscular atrophy

NMD Pharma has announced that the first patient has been dosed in its phase 2 clinical trial for its CIC-1 …

News

EC approves Roche’s Evrysdi for babies under two months with SMA

August 30, 2023
Research and Development European Commission, Evrysdi, Musculo-skeletal disorder, Roche, spinal muscular atrophy

Roche has announced that the European Commission (EC) has approved the extension of Evrysdi’s marketing authorisation in the EU to …

News
Roche building

Roche’s Evrysdi drug for SMA shown to significantly increase survival

April 15, 2021
PTC Therapeutics, Roche, pharma, pharma news, spinal muscular atrophy

Roche’s Evrysdi therapy for the treatment of children with type 1 spinal muscular atrophy (SMA) has been shown to increase …

News
Roche building

Roche’s SMA treatment Evrysdi approved by European Commission

March 30, 2021
Manufacturing and Production EMA, European Commission, PTC Therapeutics, Roche, sma, spinal muscular atrophy

The European Commission has granted marketing authorisation to Roche and PTC Therapeutics’ Evrysdi, for the treatment of patients aged 2 …

News

Roche and PTC’s SMA treatment shows positive trial results

March 16, 2021
Manufacturing and Production PTC, PTC Therapeutics, Roche, sma, spinal muscular atrophy

Roche and PTC Therapeutics’s Evrysdi treatment for children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA) …

News

NICE recommends £1.79m gene therapy for spinal muscular atrophy in babies

March 8, 2021
Sales and Marketing NICE, spinal muscular atrophy

NICE is set to approve a new and potentially curative one-off gene therapy for babies with the rare genetic disorder …

News
novartis_outside_1

Novartis unveils strong Phase 3 efficacy for one-time therapy Zolgensma in paediatric spinal muscular atrophy

October 1, 2020
Research and Development Novartis, Zolgensma, spinal muscular atrophy

Novartis has lifted the curtain on new Phase 3 data from an ongoing trial of 33 participants on the efficacy …

News
fdaoutsideweb

Genentech’s Evrysdi becomes first FDA-approved oral treatment for spinal muscular atrophy

August 10, 2020
Sales and Marketing Evrysdi, FDA, Genentech, Roche, spinal muscular atrophy

Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy …

News
novartis_outside_1

Novartis reveals hard-hitting five-year data for its gene therapy Zolgensma in spinal muscular atrophy

March 25, 2020
Research and Development Novartis, Zolgensma, gene therapy, pharma, spinal muscular atrophy

Novartis has revealed five-year data on its gene therapy Zolgensma, boasting that the one-time infusion demonstrated “rapid, significant and clinically …

News

Japan approves Novartis’ Zolgensma for spinal muscular atrophy in patients under two years old

March 19, 2020
Sales and Marketing Japan, Novartis, Zolgensma, pharma, spinal muscular atrophy

Novartis has revealed that its gene therapy Zolgensma (onasemnogene abeparvovec) has secured approval from Japan’s Ministry of Health, Labour and …

News
roche__tree

Roche’s risdiplam hits main goal in infants with spinal muscular atrophy

January 23, 2020
Research and Development Roche, pharma, risdiplam, spinal muscular atrophy

Roche has announced the success of its survival motor neuron-2 (SMN2) splicing modifier risdiplam in a new trial investigating its …

News
roche

Roche’s risdiplam hits main goal in spinal muscular atrophy type 2 or 3

November 11, 2019
Research and Development, Sales and Marketing Roche, pharma, risdiplam, spinal muscular atrophy

Roche has lifted the curtain on new data which shows that its investigational survival motor neuron-2 (SMN2) splicing modifier risdiplam …

News
novartis_outside_1

British baby’s death not caused by Zolgensma says Novartis

September 19, 2019
Research and Development AveXis, Novartis, Zolgensma, spinal muscular atrophy

Swiss drugmaker Novartis have stated that a British baby’s death this year after getting Novartis’ gene therapy Zolgensma, was not …

News

Biogen unveils promising new data for Spinraza in pre-symptomatic infants with spinal muscular atrophy

July 2, 2019
Manufacturing and Production, Research and Development Biogen, Spinraza, pharma, spinal muscular atrophy

Biogen has introduced new data on the efficacy of Spinraza in the treatment of pre-symptomatic infants in spinal muscular atrophy …

News

Latest content

Sanofi shares results for phase 3 LUNA 3 trial in immune thrombocytopenia

April 23, 2024

FDA approves ImmunityBio’s Anktiva bladder cancer treatment

April 23, 2024

EC approves Pfizer’s Emblaveo for multidrug-resistant infection treatment

April 22, 2024

Neurolentech and Kaerus Bioscience enter partnership for research on neurodevelopmental disorder

April 22, 2024

Ardena announces €20m expansion following first GMP approval

April 19, 2024