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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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

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Gene Therapy Gene Protein FDA Approval 98

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NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. — Spinal Muscular Atrophy UK (@SMA_UK_) June 2, 2021.

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Gene Therapy Gene Drugs Medicine 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. With the approval, Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy also became the first CRISPR/Cas9-based therapy approved in the US.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

pharmaphorum

NOVEMBER 16, 2022

Only a few years later, bluebird bio’s gene therapy, Zynteglo (betibeglogene autotemcel), was approved in the European market for the treatment of the rare blood disorder, beta thalassaemia. However, with a price tag of €1.58 At more than $2.1 The post Advanced therapies and the high-profile pricing dilemma appeared first on.

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