Pharm Exec12d
Biogen Seeks FDA Approval for a Higher Dosing Regimen of Spinraza for Spinal Muscular Atrophy
Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...
pharmaphorum8d
Germany first in EU to get Novartis' SMA gene therapy, costing almost 2m euros
Zolgensma can be used in patients with inherited mutations affecting genes known as SMN1, who have either been diagnosed with SMA type 1 - the most severe type - or have up to 3 copies of ...
Mid-Day on MSN4d
Budget 2025: Why no GST relief for rare disease drugs, ask parents
Parents of children with Spinal Muscular Atrophy (SMA), a rare disease, say the budget is mere eyewash. The custom duty ...
Mother in disbelief after man she met once gives van for her terminally ill son
A Wentzville mother Allyson Jercick said this van has changed her 8-year-old son's life. ST CHARLES, Mo. — We often hear about the season of giving exclusively at the end of each year, but some people ...
pharmaphorum8d
Novartis offers reimbursement options as EU backs costly SMA therapy
European approval is based on the completed phase 3 STR1VE-US and phase 1 START trials that tested the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 ...
NZ Doctor1d
Newborn screening for spinal muscular atrophy to begin next week
From February 12, all newborn babies will be offered screening for spinal muscular atrophy (SMA), a rare progressive neuromuscular disease. SMA will be one of more than 20 other rare disorders ...