Rare but Not Alone: 1 in 10 Live with a Rare Disease
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.
Raising the Profile of Rare Diseases
The rare disease community is finally getting a voice in state policy discussions thanks to a rapidly expanding movement.
Focusing on a Rare Threat for Transplant Patients
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 Disease Captures the World’s Attention
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.
Proposed Programs Could Speed Up Drug Approval for Rare Diseases
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 Offer Answers for Rare Disease Patients
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 Coverage Battle Continues for Veterans with Transthyretin Amyloidosis
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.
Economic Impact of Rare Diseases Tops $960 Billion
Rare diseases don’t just make life difficult. They also create a financial burden for patients, families, communities and the health care system.
Amyloidosis: Rare Disease, Rare Courage
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.
Is Health Care Access a Losing Battle for Veterans?
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.
