spinal muscular atrophy

News

Scholar Rock preps filings after SMA antibody clears trial

Shares in Scholar Rock have gone into overdrive after the biotech reported positive phase 3 results with apitegromab in patients with spinal muscular atrophy (SMA), setting up regulatory fi

News

Novartis opens new frontier in Voyager gene therapy alliance

Novartis has doubled down on its gene therapy collaboration with Voyager Therapeutics, stumping up another $1.2 billion-plus in potential milestone payments and directing

News

Free access to Zolgensma curbed, says Novartis

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.

News

Novartis reports two liver-related deaths with Zolgensma

Concern about the safety of gene therapies has been thrust into the spotlight again with the news that two patients treated with Novartis' spinal muscular atrophy (SMA) treatment Zolgensma

Market Access

Time is motor neurons – the importance of newborn screening ...

Spinal muscular atrophy (SMA) is a devastating, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that results in the progressive and irreversible loss of motor neuro

News

Biogen looks beyond Spinraza in SMA with Ionis deal

Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise.

Scholar Rock preps filings after SMA antibody clears trial

Novartis opens new frontier in Voyager gene therapy alliance

Free access to Zolgensma curbed, says Novartis

Novartis reports two liver-related deaths with Zolgensma

Time is motor neurons – the importance of newborn screening ...

Biogen looks beyond Spinraza in SMA with Ionis deal

Lilly grabs first-ever FDA nod for a sleep apnoea drug

The AI effect in pharma commercial strategy: From data to re...

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spinal muscular atrophy

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