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 ...
technologynetworks4d
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 ...
Armed robbery in Revesby4d
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 ...
News Medical on MSN3d
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.
Sangamo reports updated data for gene therapy candidate ST-920
Sangamo (SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate ST-920 in the treatment of Fabry disease. Read more here.
£1.65m gene-editing therapy offers hope of cure for some blood disorder patients
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...
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 ...
NHS England8d
Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients
NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as ...
GoodNewsNetwork10d
New Base Editing Gene Therapy Cures its First Patient: A Sickle-Cell Sufferer Now in the ‘Gym Every Day’
Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.
Groundbreaking £1.65m gene-editing therapy approved for NHS use offers hope to severe sickle cell patients
The UK has approximately 17,500 individuals living with sickle cell disease, a condition particularly prevalent amongst those ...