Confirmatory Part 2 of FIREFISH Demonstrates Survival and Motor Milestones Not Seen in Natural History in Infants with Type 1 Spinal Muscular Atrophy
PTC Therapeutics, Inc. (PTCT)
Last ptc therapeutics, inc. earnings: 4/30 04:01 pm
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Source: PR Newswire
SOUTH PLAINFIELD, N.J., April 28, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced positive results from part 2 of the pivotal FIREFISH study evaluating risdiplam in infants with type 1 spinal muscular atrophy (SMA). The global study met its primary endpoint of infants sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Type 1 SMA babies do not achieve this milestone without therapy. Substantial results were demonstrated across multiple secondary and exploratory endpoints, as infants in the study achieved milestones not seen in natural history. In addition to being able to sit without support, infants were able to maintain upright head control, roll to the side, and stand without support after 12 months of treatment. At 12 months, 95% infants maintained the ability to swallow and 89% were able to feed orally. No new safety signals were ident
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News
- Principal Spectrum Preferred Secs Active ETF declares monthly distribution of $0.0827 [Seeking Alpha]Seeking Alpha
- Principal Investment Grade Corporate Active ETF declares monthly distribution of $0.0792 [Seeking Alpha]Seeking Alpha
- PTC Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) [Yahoo! Finance]Yahoo! Finance
- PTC Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)PR Newswire
- PTC Therapeutics (NASDAQ:PTCT) was downgraded by analysts at Royal Bank Of Canada from an "outperform" rating to a "sector perform" rating.MarketBeat
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- 11/4/25 - Beat
PTCT
Sec Filings
- 12/4/25 - Form SCHEDULE
- 12/3/25 - Form 4
- 12/3/25 - Form 144
- PTCT's page on the SEC website