...sulfate. The disorder is caused by a mutation in the NAGLU gene; tralesinidase alfa is NAGLU...
...to in-license additional candidates in the near term. Targets IGF-2 - Insulin-like growth factor 2 NAGLU...

...sulfate. The disorder is caused by a mutation in the NAGLU gene; tralesinidase alfa is NAGLU...
...to in-license additional candidates in the near term. Targets IGF-2 - Insulin-like growth factor 2 NAGLU...

...IIIB (MPS IIIB, Sanfilippo type B syndrome). ABO-101 is an AAV9 vector that delivers the N-acetylglucosaminidase alpha (NAGLU)...
...and plasma by 60%. ABO-101 also reduced urinary total glycosaminoglycans (GAGs) by 67% and normalized NAGLU...
...Business: Endocrine/Metabolic Molecular target: N-acetylglucosaminidase alpha (NAGLU) Description: Adeno-associated viral serotype 9 (AAV9) vector that delivers the NAGLU...

...mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo B syndrome). ABO-101 is an AAV9 vector that delivers the N-acetylglucosaminidase alpha (NAGLU)...
...said ABO-101 significantly reduced cerebrospinal fluid (CSF), urine and plasma heparan sulfate levels and normalized NAGLU...
...animal studies of high-dose systemic AAV9 variants (see BioCentury Extra, Feb. 5) . Alicia Parker ABO-101 Abeona Therapeutics Inc. N-acetylglucosaminidase alpha (NAGLU)...

...AAV9 Abeona Therapeutics Inc. (NASDAQ:ABEO) ABO-101 Mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo type B syndrome) N-acetylglucosaminidase alpha (NAGLU)...

...were presented at the Inborn Errors of Metabolism meeting in Rio de Janeiro. BMN-250 is N-acetylglucosaminidase alpha (NAGLU)...
...Pharmaceutical Inc. (NASDAQ:BMRN), San Rafael, Calif. Product: BMN 250 Business: Endocrine/Metabolic Molecular target: N-acetylglucosaminidase alpha (NAGLU) Description: NAGLU...
...on brain structure assessed by MRI Status: Phase I/II data Milestone: NA Claire Quang BMN 250 BioMarin Pharmaceutical Inc. N-acetylglucosaminidase alpha (NAGLU) Inborn...

...MPS IIIB, Sanfilippo B syndrome) will continue to receive SBC-103. The product is recombinant human N-acetylglucosaminidase alpha (NAGLU)...
...May 11, 2015 ). Alexion Pharmaceuticals Inc. (NASDAQ:ALXN), New Haven, Conn. Business: Endocrine/Metabolic Chris Lieu SBC-103 Alexion Pharmaceuticals Inc. N-acetylglucosaminidase alpha (NAGLU)...

...will seek options, including partnerships, for AMT-110 and AMT-140. AMT-110, an AAV5 vector carrying the N-acetylglucosaminidase alpha (NAGLU)...
...the Netherlands Business: Gene/Cell therapy, Pharmaceuticals Chris Lieu AAV-S100A1 AAV2-GDNF AMT-060 AMT-110 AMT-130 Bristol-Myers Squibb Co. uniQure N.V. Factor IX Glial cell-derived neurotrophic factor (GDNF) Huntingtin (HTT) N-acetylglucosaminidase alpha (NAGLU) S100...

...Phase I/II candidate AMT-140 to treat Parkinson's disease. AMT-110 is an AAV5 vector carrying the N-acetylglucosaminidase alpha (NAGLU)...
...shed $0.57 to $7.73 on Tuesday. Chris Lieu AAV-S100A1 AAV2-GDNF AMT-060 AMT-110 AMT-130 Bristol-Myers Squibb Co. uniQure N.V. Factor IX Glial cell-derived neurotrophic factor (GDNF) Huntingtin (HTT) N-acetylglucosaminidase alpha (NAGLU) S100...

...Pharmaceutical Inc. (NASDAQ:BMRN), San Rafael, Calif. Product: BMN 250 Business: Endocrine/Metabolic Molecular target: N-acetylglucosaminidase alpha (NAGLU) Description: N-acetylglucosaminidase alpha (NAGLU)...