Browsing Tag
Spinal muscular atrophy
5 posts
What clinicians and regulators will watch next after Itvisma’s positive CHMP opinion
Novartis Pharma AG’s Itvisma gains CHMP backing. Discover what clinicians, regulators, and investors will watch next in SMA gene therapy.
Why Scholar Rock believes apitegromab could still become a major SMA therapy despite FDA delays
Scholar Rock targets a 2026 BLA resubmission and potential launch for apitegromab in spinal muscular atrophy. Discover what it means for the SMA market.
FDA approves Genentech’s Evrysdi tablet as first SMA treatment in tablet form
In a significant development for the treatment of spinal muscular atrophy (SMA), the U.S. Food and Drug Administration…
PTC Therapeutics collaborates with Royalty Pharma in $1.5bn Evrysdi royalty agreement
PTC Therapeutics, Inc. (NASDAQ: PTCT) has forged an agreement with Royalty Pharma plc., monetizing up to a staggering…
Novartis to acquire AveXis for $8.7bn to expand gene therapy and neuroscience portfolio
Novartis, a Swiss pharmaceutical giant, has confirmed a substantial acquisition of AveXis, a US-based gene therapy company, for…