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Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

The parents of two-year-old Aakriti Singh diagnosed with Spinal Muscular Atrophy – SMA type-1, are worried about her next ...

SMA type 1 often results in death by the age of 2 without breathing support. Life expectancy for SMA type 2 varies but is usually between 20 and 40 years. SMA types 3 and 4 typically don’t ...

More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...

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 ...

Nonadherence by SMA patients to their prescribed regimen of Evrysdi is linked to higher healthcare costs, a real-world U.S.

The most efficient one is called SMN 1 – that’s what’s missing in ... genetic testing to help determine the type of SMA, carrier testing from family members, prenatal testing before the ...

Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene ...

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 ...