The 30 million Americans diagnosed with a rare disease represent a large, underserved patient population. Their complex conditions are often poorly understood, and most have no approved treatment.
The rare disease community is finally getting a voice in state policy discussions thanks to a rapidly expanding movement.
For many patients, an organ transplant is a beacon of hope; a kidney or bone marrow transplant can be lifesaving. Sometimes, though, these very procedures introduce new, often deadly threats to their health.
A rare but devastating disease is finally getting its due. With the designation of a World Amyloidosis Day, the advocacy community makes an important move toward greater awareness.
New pilot programs to expedite the approval process for drugs that treat rare and currently untreatable diseases could be on the horizon.
Genetic testing and counseling can be critical for health care providers and patients to make informed treatment choices – especially when it comes to rare diseases.
The Veterans Health Administration exists to integrate health care services for those who served in the U.S. Armed Forces. Yet for many veterans, getting access to treatment for transthyretin amyloidosis, a rare disease caused by a buildup of abnormal proteins, is difficult.
Rare diseases don’t just make life difficult. They also create a financial burden for patients, families, communities and the health care system.
For Valerie, it began with shortness of breath and trouble sleeping. Eric found he could no longer stand in the shower. And Janet discovered she could barely hold a pen to sign her name.
Accessing proper health care can be a tough fight for America’s veterans. Those living with a rare disease called amyloidosis provide a telling example.