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5 Companies unraveling the potential of Gene Based Therapies for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type III (MPS III)

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...

MUSTANG BIO: SOARING TO A NEW HIGH AFTER BUBBLE BOY CURE ANNOUNCEMENT

Mustang Bio’s (NASDAQ: MBIO) shares rose by almost 250% after its development partner, St Jude Children’s Research Hospital in Memphis, published study results that demonstrated that its experimental gene therapy could cure “Bubble Boy” disease or X-SCID, a severe combined immunodeficiency in infants. Figure 1 - Stock...

Emerging Gene Therapy comes to the aid of hearing loss patients

Hearing impairment is a chronic disability that affects people of all age groups. Worldwide over 360 million people are affected by this condition making it the second most prevalent health issue. According to the data made available by National Institute on Deafness and Other Communication Disorders (NIDCD),...

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