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Gene therapy may be 'one-shot stop' for rare bone diseaseFor 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. "It's been a ...
Sangamo Therapeutics Inc (SGMO) reports significant progress in its Fabry disease program and neurology therapies, while ...
Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...
Nevertheless, these data led to FDA approval in November 2023 for recombinant ADAMTS13 as prophylactic or on-demand enzyme replacement therapy for the extremely rare, chronic disorder that causes ...
News Medical on MSN11d
Hard evidence of soft teeth: The oral symptoms of hypophosphatasiaGiven that there is now an enzyme replacement therapy available to treat hypophosphatasia, its early diagnosis is ...
Raymond James analyst Ryan Deschner lowered the firm’s price target on Inozyme (INZY) to $12 from $24 and keeps an Outperform rating on the ...
Five children under one year of age with the condition also known as mucopolysaccharidosis type II (MPS II) will be treated with autologous hematopoietic stem cell (HSC) gene therapy. The children ...
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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