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License Agreements - Additional Information (Detail) (USD $)
1 Months Ended 3 Months Ended 9 Months Ended 12 Months Ended 9 Months Ended 12 Months Ended 9 Months Ended 12 Months Ended 1 Months Ended 12 Months Ended 1 Months Ended
Apr. 30, 2007
Dec. 31, 2012
Sep. 30, 2012
Jun. 30, 2012
Mar. 31, 2012
Dec. 31, 2011
Sep. 30, 2011
Jun. 30, 2011
Mar. 31, 2011
Dec. 31, 2011
Dec. 31, 2010
Dec. 31, 2012
Mar. 31, 2011
Dec. 31, 2011
Licensing Agreements
Dec. 31, 2010
Licensing Agreements
Dec. 31, 2012
Licensing Agreements
Mar. 31, 2011
Licensing Agreements
Mar. 31, 2010
Licensing Agreements
License Fees
Mar. 31, 2011
Licensing Agreements
Sub License Fees
Mar. 31, 2010
Licensing Agreements
Sub License Fees
Dec. 31, 2011
Licensing Agreements
Milestone Payable
Dec. 31, 2012
Families of Spinal Muscular Atrophy
Licensing Agreements
Mar. 31, 2010
Families of Spinal Muscular Atrophy
Licensing Agreements
License Fees
Mar. 31, 2010
Families of Spinal Muscular Atrophy
Licensing Agreements
Sub License Fees
Apr. 30, 2011
Families of Spinal Muscular Atrophy
Licensing Agreements
Milestone Payable
Dec. 31, 2012
Scripps Research Institute
Licensing Agreements
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
Allocated Based on Selling Price of Deliverables
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
Transitional Services
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
Completion of Second Cohort Clinical Trial
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
Clinical Trial Material
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
Clinical Milestone Events
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
First Commercial Sale Milestone Events
Dec. 31, 2012
Pfizer Incorporation
Nonsoftware License Arrangement
First Clinical Milestone Payment
Jan. 31, 2013
Pfizer Incorporation
Nonsoftware License Arrangement
Up-front Payment Arrangement
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                                      
Payment received under license agreement                                                                     $ 5,000,000
Total future milestone receivable                                                     65,000,000         30,000,000 35,000,000 2,000,000  
License agreement, revenue recognized                                                     4,876,000 124,000 76,000 35,000 13,000        
Estimated selling price used to allocate consideration                                                         600,000 275,000 105,000        
License Agreement, initial license fee                                                   300,000                  
Total future milestone payments                                                   4,300,000                  
Common stock, shares issued                                                   87,464                  
Common stock, shares issued value                                                   300,000                  
Common stock, shares purchased through issuance of warrants 150,000                                                 150,000                  
Warrant term 7 years                                                 7 years                  
License Agreement termination, written notice period                                                   90 days                  
Research and development expenses   2,343,000 2,433,000 2,906,000 2,808,000 2,870,000 3,075,000 3,517,000 3,785,000 9,461,960 8,744,548 10,489,811 12,528,819 525,000 343,000 55,000 374,000 500,000 65,000 175,000 500,000   500,000 175,000 500,000                    
Research and development, financial obligation                               $ 16,000,000           $ 16,000,000                          
License Agreement, expiration term                                           10 years