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spinal muscular atrophy

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

to children with spinal muscular atrophy (SMA).

UK government allocates up to £680m a year to two funds offering cutting-edge treatments

decisions. Over the past year, NHS England has negotiated contracts for a range of new treatments, including Zolgensma for spinal muscular atrophy (SMA).
Genentech’s Evrysdi gets extended FDA approval for spinal muscular atrophy

Genentech’s Evrysdi (risdiplam), a treatment for spinal muscular atrophy (SMA), has been given an extended approved by the US Food and Drug Administration (FDA) to include babies under two months
SOLVE FSHD announces the appointment of Dr Eva Chin as its executive director

It was also shared that Chip Wilson, founder of SOLVE FSHD, has committed a personal pledge of $100m towards a cure for facioscapulohumeral muscular dystrophy (FSHD), a rare disease he was ... Dr Chin has been responsible over the last five years for
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).
FDA grants priority review for Roche’s Evrysdi for spinal muscular atrophy in babies

The review of Evrysdi has been developed for pre-symptomatic babies under two months old with spinal muscular atrophy. ... symptomatic babies diagnosed with spinal muscular atrophy (SMA) under two months of age.

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20 for 2020 – Five pharma leaders to watch in 2020

Hudson had a distinguished career at Novartis, presiding over the launches and roll-outs of psoriasis blockbuster Cosentyx (secukinumab), spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec) and heart failure therapy
The changing nature of approvals – what does the future hold?

Vision for the future. As we look forward, sustainability will continue to influence company strategies, particularly given that some new drugs, such as Zolgensma for spinal muscular atrophy (SMA), cost US$2m
Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019

the genetic defect underlying spinal muscular atrophy (SMA).
Drug launches to watch in 2019

The next step in this process is the launch of Zolgensma (AveXis) – the gene therapy for spinal muscular atrophy (SMA) which is now filed with the FDA and EMA, and is
No incentive for a cure

And Spinraza from Biogen, the first ever disease-modifying treatment for spinal muscular atrophy (SMA), which increases the body’s ability to produce SMN protein critical to the health of motor

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Getting it Right First Time: Building a Value-Based Contract Strategy

than anticipated, something we have seen even with the highly effective Zolgensma for Spinal Muscular Atrophy.
Rare Disease Day – looking to the future with hope

From the mother who spends hours at a clinical study site while her 3-year-old daughter receives an experimental treatment for spinal muscular atrophy, resolutely hopeful that she will someday ... Founded by Rich Horgan, whose brother suffers from
Article: Market access in Brazil

All eyes on Spinraza. Director Rachel Howard asks, “What does Brazil’s unprecedented risk-sharing agreement in Spinal Muscular Atrophy (SMA) mean for the future of market access in Brazil? ... In April 2019, Brazilian Health Minister Luiz Henrique

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Country Manager, Healthcare Communications Agency, Germany: Highly competitive salary

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Sciterion is an award winning specialist healthcare communications consultancy within the Havas Health network of companies. We exist to make...

The communication challenge of helping he next generation to be healthier: As the pressure on the health service increases the health of the next generation is vital for everyone. How do we show positive attitudes and change behaviour while future proofing...
Are we losing sight of what the democratisation of healthcare really looks like?: We have a core responsibility as healthcare communicators to consistently drive for better inclusion, engagement and compliance. What does the ‘democratisation of healthcare’ really look like?...
How the Royal Society’s EiR programme can help to drive UK innovation: ...

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