Pre-symptomatic infants with spinal muscular atrophy (SMA) treated with Evrysdi were able to reach motor milestones such as sitting without support, rolling, crawling, standing unaided and walking independently, according to PTC Therapeutics Inc, announcing positive interim results from the ongoing RAINBOWFISH trial.

“It is incredible to see that pre-symptomatic SMA infants are able to achieve developmental milestones consistent with healthy children. These results broaden our knowledge of Evrysdi in babies younger than two months of age and adds to the proven efficacy data in a broad range of SMA patients.” 

— Stuart W. Peltz, PhD, Chief Executive Officer, PTC Therapeutics

Additionally, results presented from a 12-month interim analysis of the JEWELFISH safety study demonstrated overall stabilization in motor function and rapid and sustained increases in SMN protein. These patients began treatment with Evrysdi following previous treatments, including Spinraza or Zolgensma and are representative of the real-world, broad and heterogeneous SMA population with a high degree of motor impairment at baseline.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi has been approved for the treatment of patients with SMA, aged 2 months and older by the FDA and the EMA. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group, a media release from PTC Therapeutics Inc explains.

[Source(s): PTC Therapeutics Inc, PR Newswire]

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