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RAINBOWFISH Results Highlight Risdiplam as Effective Treatment for Presymptomatic SMA

After 1 year of treatment with risdiplam, 81% of the 26-infant cohort was able to sit independently for 30 seconds, a milestone not typically reached in spinal muscular atrophy (SMA).

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Roche presents positive results for spinal muscular atrophy treatment Evrysdi in babies

Roche presents positive results for spinal muscular atrophy treatment Evrysdi in babies
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Babies with spinal muscular atrophy able to sit up after 1 year of Evrysdi treatment

Babies with spinal muscular atrophy able to sit up after 1 year of Evrysdi treatment
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NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society

NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society
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REGENXBIO Inc. (NASDAQ:RGNX) Q2 2023 Earnings Call Transcript

Operator: Good day, and thank you for standing by. Welcome to the Q2 2023 REGENXBIO Earnings Conference Call. At this time, all participants are in a listen-only mode.

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