Please login to the form below

Not currently logged in
Email:
Password:

type 1 spinal muscular atrophy

This page shows the latest type 1 spinal muscular atrophy news and features for those working in and with pharma, biotech and healthcare.

Roche’s Evrysdi improves survival rates for infants with SMA

Roche’s Evrysdi improves survival rates for infants with SMA

FIREFISH evaluated the efficacy and safety of Evrysdi (risdiplam) in infants aged 1-7 months at the time of enrolment with type 1 spinal muscular atrophy (SMA). ... Without the treatment, children with type 1 SMA are not able to sit without support.

Latest news

  • Novartis aims for 10 blockbuster launches by 2020 Novartis aims for 10 blockbuster launches by 2020

    The one-time, potentially curative treatment for spinal muscular atrophy (SMA) has attracted a great deal of interest ahead of its approval, thanks in part to Novartis saying the treatment could ... Zolgensma – spinal muscular atrophy (SMA) type 1. 2020

  • FDA fast tracks Novartis’ SMA gene therapy FDA fast tracks Novartis’ SMA gene therapy

    Novartis has announced that the FDA is to fast-track its investigational gene therapy AVXS-101 candidate for spinal muscular atrophy (SMA) type 1, meaning it could be approved as early ... Its president David Lennon, said: “This important step by the

  • Novartis: pipeline will generate 60 filings in three years Novartis: pipeline will generate 60 filings in three years

    Already active in this area with its lymphoma and leukaemia CAR-T therapy Kymriah (tisagenlecleucel), Novartis is highlighting new projects today including gene therapy AVXS101 for spinal muscular atrophy (SMA) type ... 1 – acquired as part of its

More from news
Approximately 1 fully matching, plus 3 partially matching documents found.

Subscribe to our email news alerts

Featured jobs

PMHub

Add my company
VISFO

A team of strategic decision specialists with over 30+ years in the pharma space, we give you confidence through experience....

Latest intelligence

Our Top 5 Pharma Picks From Cannes Lions
Here are five of Art's top five pharma and healthcare picks from Cannes Lions 2022....
The Evolving Role of the Sales Rep
The COVID-19 pandemic sparked an evolution in physician engagement. Jill Padgett, EdD, Head of Training, explores what this means for the sales force and shares her advice for succeeding in...
Measure your omnichannel maturity with our new tool
Try our Omnichannel Maturity Tool to gain tangible advice and an instant analysis of your omnichannel readiness....