New gene therapy modalities, such as CRISPR guide RNA (single guide ribonucleic acid [sgRNA]) and messenger RNA (mRNA), ...

Discover Regional Growth Trends and Shares for Galsulfase, Velaglucerase Alfa, Laronidase, Asfotasealfa, and Other ERT ...

AceLink is advancing a small-molecule treatment option for Fabry disease, which could provide a more convenient alternative ...

Researchers from the Nagoya University Graduate School of Medicine identified an enzyme, alpha-kinase 2 (ALPK2) that is ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

The global enzyme replacement therapy market size was valued at USD 10.12 billion in 2024 and is projected to grow from USD 11.35 billion in 2025 to reach USD 21.29 billion by 2033, exhibiting a CAGR ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...

After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

University of Illinois Chicago scientists have redesigned a treatment for the most common pediatric leukemia to eliminate its severe side effects, like blood clots and liver damage. If approved, the ...

Stifel analysts confirmed their Buy rating on Denali Therapeutics Inc. (NASDAQ:DNLI) with a steady price target of $37.00. According to InvestingPro data, analysts maintain a Strong Buy consensus with ...