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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 chooses internal replacement for US pharma president role

of the spinal muscular atrophy (SMA) gene therapy, but did not disclose its findings until after it received marketing approval.

Latest news

ICER closely monitoring FDA’s Zolgensma investigation

Novartis’ gene therapy Zolgensma was approved by the FDA to treat spinal muscular atrophy (SMA) in children under two years of age earlier this year.
Novartis defends Zolgensma as FDA investigates ‘data manipulation’

Gene therapy will remain on the market. Novartis withheld data prior to the approval of Zolgensma, its spinal muscular atrophy (SMA) gene therapy, the US Food and Drug Administration (FDA) has ... It was also the first gene therapy approved for
New products help lift Roche outlook despite biosimilar onslaught

Severin Schwan. One of the firm's most promising late-stage pipeline projects is spinal muscular atrophy (SMA) project risdiplam, which is set to rival Biogen’s Spinraza.
Biogen grows in Q2, but Spinraza performance a concern

Spinraza is Biogen’s spinal muscular atrophy (SMA) treatment, and William Blair had forecast sales of the period at $540m, but it could only reach $488m.
FDA will maintain the pace, vows Woodcock, but warns of skills gap

Also eyecatching were Woodcock’s comments on two high profile FDA approvals of spinal muscular atrophy (SMA) treatments, Biogen’s Spinraza and Novartis’ gene therapy Zolgensma.

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Latest Intelligence

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.
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.
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
No incentive for a cure

to 12-week course. 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.
Deal Watch July 2016

In 2014 the partners expanded the agreement for CK-2127107 to include some neuromuscular indications, such as Spinal Muscular Atrophy (SMA).

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Latest appointments

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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