for the treatment of mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. MPS II is a rare, X-linked recessive disorder caused by lysosomal iduronate-2-sulfatase (I2S) enzyme ...

About MPS II MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans (GAGs), including ...

The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder. Five ...

was approved in Japan for the treatment of mucopolysaccharidosis II (MPS II or Hunter syndrome). MPS II is a rare, inherited disorder caused by defects in iduronate-2-sulfatase (IDS), an enzyme ...

Thursday, May 15, is MPS Awareness Day, a time to spread the word about this group of ultra rare diseases. When Jamestown ...

for RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The regulator has granted priority review for the BLA with a Prescription Drug User Fee Act ...

DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter syndrome (MPS II).

s (NASDAQ: RGNX) Biologics License Application (BLA) for clemidsogene lanparvovec, known as RGX-121, aimed at treating Mucopolysaccharidosis II (MPS II), or Hunter syndrome. The agency has set a ...

About MPS II MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans (GAGs), including ...