Disease Tab: Bone

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A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)

Rocket Pharmaceuticals is conducting a clinical trial using a gene therapy for infantile malignant osteopetrosis (IMO), a rare and life-threatening disorder that develops in infancy.  IMO is caused by defective bone cell function, which results in blindness, deafness, bone marrow failure, and death very early in life.  The trial will use a gene therapy that targets IMO caused […]

Evaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis

Researchers at the California Institute for Biomedical Research (CALIBR) have been awarded $8.447 million to test KA34, a drug that, in preclinical tests, recruits stem cells to create new cartilage in areas damaged by osteoarthritis. CIRM funded the research that developed this technology and now this Phase 1 trial will test this stem cell directed […]

Treatment of non-traumatic osteonecrosis with endogenous Mesenchymal stem cells

Femoral head osteonecrosis (aka avascular necrosis) is a disease caused by loss of blood supply to the bone, leading to bone cell death, end stage hip arthritis and total hip replacement. There is an unmet need for treatment of this disease, that affects individuals at prime of life (peak age 35 years). This small molecule […]