pharmaphorum

JANUARY 9, 2023

Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

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119

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

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pharmaphorum

JUNE 2, 2021

NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. “NICE’s initial ‘no’ to approving risdiplam for NHS funding is not unexpected,” said SMA UK this morning.

Documentation 52

Documentation Communication 52

European Pharmaceutical Review

MARCH 1, 2023

This would also provide a template to allow the proteins to align correctly and form crystals. To create the surface required, the team coated gold nanoparticles with bioconjugates maleimide (MAL) and N -hydroxysuccinimide ester (NHS). And because the proteins are aligned, they can form a crystal faster.

Vaccines 93

Vaccines 93

pharmaphorum

DECEMBER 23, 2022

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Results suggest durable epigenetic re-wiring and provide rationale for intermittent clinical dosing. Professional reception of the findings.

Drug Development 52

Drug Development Immunity Immunization Hospitals 52