Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
Enzyme Replacement Therapy Market Trends, Developments and Strategies 2025-2030: A Projected $15.44 Billion Landscape
Discover Regional Growth Trends and Shares for Galsulfase, Velaglucerase Alfa, Laronidase, Asfotasealfa, and Other ERT ...
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AceLink’s Fabry Hopeful Shows Potential in Mid-Stage Study
AceLink is advancing a small-molecule treatment option for Fabry disease, which could provide a more convenient alternative ...
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New gene therapy shows promise for lifelong treatment of hypophosphatasia
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.
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Gene Therapy Shows Promise for Rare Bone Disease in Mice
Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...
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Denali eyes approval after Hunter syndrome drug clears safety measures, reduces biomarkers in open-label study
After winning a breakthrough therapy designation for its Hunter syndrome enzyme replacement therapy, Denali Therapeutics is ...
uniQure advances gene therapy trial for Fabry disease
LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...
Enzyme Replacement Therapy Market Size, Growth & Forecast By 2033
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 ...
Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease
FDA aligned on single-arm Phase 3 study to support potential accelerated and full approvalOn track to dose first patient in Phase 3 trial in ...
Science Daily9d
Gene therapy may be 'one shot stop' for rare bone disease
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. Currently ...
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Gene Therapy: One-Shot Cure for Rare Bone Disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by ...