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SMA
This page shows the latest SMA news and features for those working in and with pharma, biotech and healthcare.
Novartis hit by ‘COVID-19-related lockdowns’ in Q4
added. In the Q4 results, Novartis highlighted its main growth drivers – including its heart failure drug Entresto (sacubitril/valsartan), its immunology therapy Cosentyx (secukinumab) and its spinal muscular atrophy (SMA) gene
Latest news
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Biogen advances Spinraza study in SMA patients treated with Novartis’ Zolgensma
It will be conducted at around 20 sites globally, with researchers aiming to enrol up to 60 children with SMA. ... to develop SMA type 1, and who were six months old or younger when they received Zolgensma.
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Novartis weathers the COVID-19 storm in Q3, lifts outlook
Novartis’ main growth drivers, and protectors against the continuing impact caused by the COVID-19 pandemic, includes its spinal muscular atrophy (SMA) gene therapy Zolgensma and chronic heart failure med Entresto
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Bayer pays an initial $2bn for gene therapy firm AskBio
In addition, some of the research undertaken at AskBio was used to in the development of Novartis’ spinal muscular atrophy (SMA) gene therapy Zolgensma.
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Roche scores FDA approval for SMA drug risdiplam, now named Evrysdi
SMA is a muscle-wasting disease that affects around one in 6, 000 to 10, 000 live births and is a leading cause of genetic death in infants. ... The first drug to reach the market for SMA was Biogen’s Spinraza (nusinersen) back in 2016.
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Biogen plans Spinraza study in SMA patients treated with Novartis’ Zolgensma
Both companies are facing upcoming competition from Roche’s rival drug risdiplam, which has scored an FDA priority review in SMA. ... Roche is also currently investigating risdiplam in an additional clinical trial in infants with SMA type 1.
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Approximately 17 fully matching, plus 57 partially matching documents found.
Latest Intelligence
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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
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Orphan drugs and breakthrough therapies drive Cortellis Drugs to Watch 2019
the genetic defect underlying spinal muscular atrophy (SMA). ... In the third quarter of 2018, AveXis filed Zolgensma for approval in the US, EU and Japan for the treatment of SMA type I.
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Bluebird: on a mission to ‘recode’ the DNA of healthcare
Bluebird is not the only company likely to break through this 1m barrier in 2019 – Novartis’ SMA gene therapy Zolgensma is also on the cusp of approval in the US.
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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 ... The company argues that current ten- year costs of caring for SMA type 1 patients
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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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Latest appointments
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Warp Drive Bio names Gregory Verdine as CEO
He has also served as a global consultant for Roche, scientific adviser to Vertex and Ariad, and currently sits on the boards of the US National Cancer Institute and the SMA
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Latest from PMHub
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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
More from PMHub
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COVID-19 Updates and Daily News
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- STEM Healthcare founder Rob Wood secures a 25% stake in performance-io
- Sinopharm’s COVID-19 vaccine scores approval in China
- FDA authorises flexible storage for Pfizer/BioNTech COVID-19 vaccine
- UK study finds Pfizer/BioNTech, AZ/Oxford vaccines reduce severe COVID-19 in older people
- AZ/Oxford University vaccine shows 82.4% efficacy with extended dosing schedule
- Roche, Genentech partner with The Weill Neurohub on ten-year neuroscience collaboration
- Amgen adds to oncology pipeline with $1.9bn Five Prime Therapeutics acquisition
- GSK, Vir halt enrolment of phase 3 COVID-19 antibody study
- Germany backs use of AZ/Oxford COVID-19 vaccine in over-65s
- CureVac signs manufacturing agreement with Novartis for COVID-19 vaccine candidate
- #DemandDiversity: For International Women's Day, we ask... why do women often suffer from more side effects than men?
- Gene Editing and Precision Medicine: The Future of Healthcare?
PMHub
Windrose Consulting Group
Windrose Consulting Group helps life science companies maximize the commercial value of their products and bring new innovations to market...
Latest intelligence
- #DemandDiversity: For International Women's Day, we ask... why do women often suffer from more side effects than men?
- Women are largely prescribed exactly the same treatment regimens as men, with no account for the underlying differences in physiology and drug metabolism between the sexes....
- Good design saves lives
- Good design and creative thinking are essential if we are to improve on existing problems in new ways, which is why design and creativity within healthcare is vital. Health is...
- Why you must understand the pricing of patient recruitment companies
- Recruiting a diverse range of patients and engaging with them for your clinical trial isn’t an easy task, which means you might turn to patient recruitment companies, like us, who...
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