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找到 67,387 条关于 “Sanfilippo syndrome type C”

Phoenix Nest Secures $1.49 Million NIH Grant to Advance Gene Therapy Manufacturing for Sanfilippo Syndrome Type C

Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A

FDA Approves Fayuvi, First-Ever Gene Therapy for Sanfilippo Syndrome Type A

Spruce Biosciences' TA-ERT Receives FDA Breakthrough Therapy Designation for Ultra-Rare Sanfilippo Syndrome Type B

JR-446 Receives Japanese Orphan Drug Designation for Rare Sanfilippo Syndrome Type B

Spruce Biosciences Secures $50M to Advance Sanfilippo Syndrome Type B Therapy Toward BLA Submission

FDA Issues Complete Response Letter for Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A

Spruce Biosciences Reports Six-Year Data Showing Tralesinidase Alfa Preserves Cognitive Function in Sanfilippo Syndrome Type B

Spruce Biosciences Reports Breakthrough Long-Term Data for TA-ERT in Rare Sanfilippo Syndrome Type B

Spruce Biosciences Secures $50 Million Financing to Advance First Potential Treatment for Sanfilippo Syndrome Type B

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