January 6, 2022
The rare disease community is finally getting a voice in state policy discussions thanks to a rapidly expanding movement.
December 14, 2021
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.
October 26, 2021
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.
October 1, 2021
New pilot programs to expedite the approval process for drugs that treat rare and currently untreatable diseases could be on the horizon.
September 29, 2021
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.
September 29, 2021
Genetic testing and counseling are useful tools for patients with rare diseases. Sophisticated genetic tests available in clinical settings now…
July 27, 2021
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.
April 26, 2021
Watching a patient eat. Asking about family photos on the nearby coffee table. Observing the living space. These are all benefits of telemedicine that help doctors see their patients through a new lens.
March 30, 2021
Rare diseases don’t just make life difficult. They also create a financial burden for patients, families, communities and the health care system.
February 26, 2021
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.