Zolgensma shows promise in presymptomatic SMA patients
Children treated with gene therapy prior to symptom presentation achieved motor milestones not seen in the natural history of SMA
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Tag: SMA
NHS England agrees deal with Novartis for access to SMA gene therapy Zolgensma
NHS England has secured drug for use at a ‘substantial’ confidential discount
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Novartis’ Zolgensma shows ‘continued benefit’ in SMA patients
66.7% of patients in the intent-to-treat population were able to feed orally without the need for feeding support
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EU regulators to review Evrysdi for SMA
The decision triggers a £15m milestone payment from Roche to PTC Therapeutics
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Roche’s SMA treatment Evrysdi bags US approval
Drug cleared for use in SMA patients aged two months or older
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EU approves Novartis’ SMA gene therapy Zolgensma
Discussions with EU governments and reimbursement agencies are ongoing to agree on terms of an innovative ‘Day One’ access programme
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Roche’s risdiplam shows promise in Type 1 SMA
Trial findings are particularly encouraging given that infants already had progressed disease
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Five-year Zolgensma data shows SMA benefit
If left untreated, SMA type I leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.
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Zolgensma wins FDA green light in child SMA
The $2.1 milllion per treatment price tag has garnered substantial criticism.
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New data reinforce effectiveness of Novartis’ Zolgensma for SMA
The Phase III STR1VE data showed that 21 patients were alive and event-free as of September 27 last year.
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Novartis’ SMA therapy gets US priority review
US regulators have accepted for review Novartis’ Biologics License Application for Zolgensma, an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1.
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Draft guidelines reject NHS funding for Biogen’s Spinraza
The National Institute for Health and Care Excellence is not recommending NHS use of Biogen’s Spinraza for the rare genetic disorder spinal muscular atrophy (SMA) at this time.
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