April 21, 2022
What stands between rare cancer patients and life-saving treatment? A 3-to-7-character alpha-numeric code.
February 28, 2022
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.
February 10, 2022
By Johana R. Fajardo, DNP, ANP-BC - I walk with many amyloidosis patients throughout their journey, from diagnosis to end of life.
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.