Two new studies funded in part by NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) describe efforts to develop stem cell-based approaches for treating Epidermolysis Bullosa (EB), a rare, genetic skin disease. The findings, which were published jointly in the journal, Science Translational Medicine, may lead to individualized therapies for EB, and possibly other genetic diseases. People with EB have skin that is so fragile that the slightest friction causes blisters. The severity of the disease ranges from limited tearing of skin on the hands and feet to widespread blistering and scarring, including mucosal surfaces like the
What is rheumatoid arthritis? Rheumatoid arthritis (RA) is a chronic (long-lasting) disease that mostly affects joints, such as the wrist, hands, feet, spine, knees, and jaw. In joints, RA causes inflammation that leads to: Pain. Swelling Stiffness. Loss of function. Rheumatoid arthritis is an autoimmune disorder because the immune system attacks the healthy joint tissues. Normally, the immune system helps protect the body from infection and disease. RA may cause you to feel unusually tired, to have occasional fevers, and to have a loss of appetite. It also may cause other medical problems in the heart, lungs, blood, nerves, eyes
What is epidermolysis bullosa? Epidermolysis bullosa is a group of rare diseases that cause fragile skin that leads to blisters and tearing. Tears, sores, and blisters in the skin happen when something rubs or bumps the skin. They can appear anywhere on the body. In severe cases, blisters may also develop inside the body. The symptoms of the disease usually begin at birth or during infancy and range from mild to severe.
What is Marfan syndrome? Marfan syndrome is a genetic disorder that affects the body’s ability to make healthy connective tissue, which supports the bones, muscles, organs, and tissues in your body. The condition can affect different areas of the body, including: Bones, ligaments, tendons, and cartilage. Organs, such as the heart and lungs. Skin.
What is Paget’s disease? Paget’s disease of bone is a chronic (long-lasting) disorder that causes bones to grow larger and become weaker than normal. Usually only one or a few bones have the disease. Many people with Paget’s disease do not have symptoms. However, the bone changes can cause: Bone pain. Misshapen bones. Broken bones (fractures). Problems in the joints near the bones with the disease. With treatment, many people can: Manage their symptoms. Improve pain. Control the effects of the disease.
Media Availability What: The U.S. Food and Drug Administration recently approved a new oral medication for the treatment of rheumatoid arthritis that represents a new class of drugs for the disease. The drug, tofacitinib (Xeljanz), provides a new treatment option for adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to, or who are intolerant of, methotrexate, a standard therapy for the disease. Affecting nearly 1.5 million adults, rheumatoid arthritis is an inflammatory disease that causes pain, swelling, stiffness, and loss of function in the joints. It occurs when the immune system, which normally defends
The AMP RA/SLE Program brings together the NIH and medical community to address rheumatoid arthritis and lupus with diagnostic and drug advancements.