1180 CELEBRATION BOULEVARD, SUITE 103, CELEBRATION, FL
Earnings Release
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Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $150 Million
Reports Fourth Quarter and Full Year 2024 Financial Results
Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million
Announces Organizational Changes
U.S. Commercial Availability of MIPLYFFA™ (arimoclomol) for Treatment of Niemann-Pick Disease Type C
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FY 2024
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FY 2023
Registration Statement for Securities to be Offered to Employees
Prospectus filed pursuant to Rule 424(b)(5)
Prospectus Filed Pursuant to Rule 424(b)(2)
Effectiveness Notice
Registration Statement for Securities Offered under a Shelf Registration
Prospectus filed pursuant to Rule 424(b)(3)
Additional Proxy Materials
Contested Proxy Solicitation Materials
PRRN14A
Definitive Additional Information Statement
Statement of Changes in Beneficial Ownership
Notice of Proposed Sale of Securities
Correspondence
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