v3.26.1
Revenue, net - Narrative (Details) - USD ($)
3 Months Ended 12 Months Ended
Mar. 31, 2026
Mar. 31, 2025
Dec. 31, 2025
Sep. 03, 2019
Disaggregation of Revenue [Line Items]        
Revenue, net $ 36,220,000 $ 20,401,000    
Contract with customer, liability 0   $ 0  
Reserve or allowance for doubtful accounts 0   0  
Arimoclomol Purchase Agreement        
Disaggregation of Revenue [Line Items]        
Recognized revenue 10,200,000 2,300,000    
Clawback liability 4,700,000 1,700,000    
Estimated reserve liability 16,900,000   15,300,000  
MIPLYFFA        
Disaggregation of Revenue [Line Items]        
Revenue, net 24,600,000 17,100,000    
OLPRUVA        
Disaggregation of Revenue [Line Items]        
Revenue, net 300,000 100,000    
License | AZSTARYS License Agreement        
Disaggregation of Revenue [Line Items]        
Recognized revenue   $ 900,000    
Contract with customer, liability, revenue recognized, including opening balance 1,100,000      
Contract with customer, liability $ 0   $ 0  
License | Commave Therapeutics SA | AZSTARYS License Agreement        
Disaggregation of Revenue [Line Items]        
Milestone payments       $ 590,000,000.0
License | Relief Therapeutics        
Disaggregation of Revenue [Line Items]        
Royalty percentage 10.00%