When she was just ten months old, Elizabeth Lobato was diagnosed with a severe form of osteogenesis imperfecta (OI), also known as brittle bone disease. Ms. Lobato’s head was not fully hardened when she was born and her bones were frail. This rare genetic disorder leads to muscle weakness, hearing loss, loose joints, curved bones, scoliosis and brittle teeth. Many children eventually die due to dangerous respiratory problems and infections. As a child, the native Californian suffered breaks and fractures to her legs, ribs and other parts of her body. She stopped growing sometime around the age of five and, at eleven, she was just two and a half feet tall. Her body was extremely fragile and she had to receive regular hospital care.

In 2010, at the age of eleven, Ms. Lobato began receiving bone marrow-derived stem cells from her father as part of an investigational OI study being conducted by Dr. Edwin Horwitz, a bone marrow and stem cell researcher at Children’s Hospital of Philadelphia. The stem cells appear to have a crucial regenerative trait: when released into the bloodstream, they have shown signs of forming new bone, cartilage and fat cells. She received the stem cell infusions every four months for four years and soon grew to three feet four inches. Her head hardened and since 2011, she has not had any fractures. Her quality
of life greatly improved as well. She is now able to be
 on her own and does not get injured as easily. In June 2016, Ms. Lobato is scheduled to receive more infusions of her father’s stem cells at Nationwide Children’s Hospital in Columbus, Ohio, where Dr. Horwitz is now director of Blood and Marrow Transplant.

At seventeen, Ms. Lobato epitomizes the unwavering hope for a better life that many people with rare genetic disorders have. She is now an OI spokesperson and awareness advocate and wishes that no child has to suffer like she did. She is scheduled to attend college in September 2016. Her parents describe her as remarkably happy and positive. Ms. Lobato enjoys astrophotography and plans to pursue her dream of being a pre-school teacher.