NVS reports new safety and efficacy data from the phase III program for its investigational OAV101 IT gene therapy for SMA ...

Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to ...

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

Detailed price information for Pacific Biosciences (PACB-Q) from The Globe and Mail including charting and trades.

Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing severe muscle weakness and hypotonia. Babies with SMA often struggle to meet ...

The kit targets genes like CFTR, CYP21A2, F8 inversions, FMR1, GBA, HBA1, HBA2, HBB, SMN1, SMN2 and TNXB. Per a report by TechSci Research, the global Nanopore Sequencing market was valued at $330 ...

The early to mid-stage dose-ranging study evaluated the safety and efficacy of the candidate in patients with SMA with three copies of SMN2 aged 6 months to less than 60 months. The OAV101 IT ...

Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...

These regions contain hundreds of genes critical to human health—including those implicated in spinal muscular atrophy (SMN1/SMN2), congenital adrenal hyperplasia (CYP21A2), and red-green color ...