None

None

None

None

AAV Gene Therapy Study for Subjects With PKU

This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency. The study consists of a 6 month randomized, double blind, placebo-controlled period, followed by 1 year of …

Gene Therapy for X Linked Severe Combined Immunodeficiency

A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCIDsevere combined immune deficiency .The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number …

Trial of Inhaled Anti-viral (SNG001) for SARS-CoV-2 (COVID-19) Infection

It has been shown that older people and people with some chronic diseases have an IFN- deficiency. Many viruses inhibit IFN- as part of their strategy to evade the immune system. Addition of IFN- in vitro protects lung cells from viral infection.

Tissue-engineered Skin Graft Repair of Autologous Scar Dermal Scaffolds

It affects the appearance and some scar contracture often leads to joint dysfunction.Patients have low quality of life, long treatment cycle, heavy social burden and high medical costs.Skin grafting is currently the gold standard for scar repair.However, there are often insufficient skin sources, easy to scar recurrence, lack of skin …

Effect of Preoperative Intervention With Folic Acid and Vitamin B12 on Postoperative Neurobehavioral Changes in Children

Comparison of preoperative folic acid and VitB12 intervention on postoperative delirium and long-term neurobehavioral changes in children under general anesthesia