14-year-old author with Spinal Muscular Atrophy inspires others with her resilience and determination
Keya Hatkar, a young author and motivational speaker with Spinal Muscular Atrophy, inspires others with her resilience and ...
Experts seek affordable access to medicines for treating Spinal Muscular Atrophy
Haris Beeran urges government intervention to make affordable SMA medicines available in India, addressing the high costs and patent issues.
MP Haris Beeran calls for local production of generic drug variant for spinal muscular atrophy
Upper House member Haris Beeran has said the current costs of medicines approved for SMA are 'beyond the reach of the central ...
The Financial Express13h
Novartis gene therapy poised to expand SMA treatment landscape
Novartis has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully ...
PharmiWeb10h
Novartis gene therapy poised to expand SMA treatment landscape, says GlobalData
Novartis has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully achieved the primary endpoint in a Phase III STEER study (NCT05089656) ...
Free SMA treatment for all to cost Rs 40,000 crore a year, not SC's call: Government
Centre informed Supreme Court that providing free gene therapy for Spinal Muscular Atrophy (SMA) could cost India Rs ...
Positive data could expand use of Novartis’ gene therapy for SMA
On 30 December 2024, Novartis reported that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) reached ...
Findlay boy provides comfort for Nationwide Children’s Hospital
He spent time at Nationwide Children’s Hospital. In November of 2023, Jackson was told he was going home. He thought this ...
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease This is the 2nd ...
Kellogg, WVU women’s basketball celebrate young fan pregame
Prior to Saturday’s women’s basketball game between West Virginia and BYU, the WVU women’s basketball team invited a young ...
GlobalData on MSN1d
FDA grants ODD to NMD Pharma’s Charcot-Marie-Tooth disease therapy
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to NMD Pharma’s oral lead development ...
FiercePharma5d
After delay with FDA, Novartis touts pivotal trial win for intrathecal Zolgensma in older SMA patients
More than three years after Novartis’ plan for a speedy approval for intrathecal Zolgensma was thwarted by an FDA requirement ...