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Congressional Hill Briefing, Improving Care for Veterans with Rare Diseases: Establishing a National Commission

We, with other members of the amyloidosis community, were proud to join an important meeting hosted by the Center for Patient Advocacy Leaders (CPALs). They hosted a Congressional Hill Briefing, Improving Care for Veterans with Rare Diseases: Establishing a National Commission, at the U.S. Capitol Visitor Center in Washington, DC. This briefing was designed to bring together Veterans/Veterans’ advocates, rare disease advocates, patients, and congressional staff to address unmet needs of Veterans with rare diseases and explore collective action to help ensure Veterans with rare diseases get the comprehensive, patient-centered care and treatment they need and deserve.

Mackenzie’s Mission at the Bradley Z Naifeh Amyloidosis Conference 2024!

We were proud to be part of the 2nd annual Bradley Z. Naifeh Amyloidosis Conference 2024 at Houston Methodist! On day 1 the auditorium was filled with healthcare professionals who learned about the many facets of the clinical side of amyloidosis from experts such as Dr. Ron Witteles of Stanford and Dr. Angela Dispenzieri of Mayo Clinic.

On day 2 the auditorium was packed with patients and caregivers of all types of amyloidosis, offering a wonderful opportunity to learn about the disease, resources available, and network with other patients and caregivers.

Mackenzie and Deb had the chance for a photo op with Megan Fleischfresser Naifeh (daughter of Bradley Z. Naifeh who lost his battle with AL Amyloidosis) and Dr. Arvind Bhimaraj (co-host of the conference).

We thank the Naifeh family for their support of this valuable annual conference.

ASB Participates in Rare Disease Day 2024!

The Amyloidosis Speakers Bureau was thrilled to participate on Rare Disease Day! One way we were part of the celebration was for our patient educator Sean to share his journey at Keck Graduate Institute’s Rare Disease Awareness Fair.

 

Breaking News – FDA approves WAINUA for ATTRv-PN

The U.S. FDA has approved a new treatment for adults living with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN).

WAINUA™ (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis.
  • U.S. FDA approval based on Phase 3 NEURO-TTRansform results showing WAINUA demonstrated consistent and sustained benefit halting neuropathy disease progression and improving neuropathy impairment and quality of life
  • Additional regulatory reviews for WAINUA underway in rest of world
  • WAINUA will be available in the U.S. in January 2024

READ PRESS RELEASE HERE

“Many people living with hereditary transthyretin-mediated amyloid polyneuropathy are unable to fully enjoy their lives because of the relentless, progressive and debilitating effects of the disease,” said Michael J. Polydefkis, M.D., professor of neurology at Johns Hopkins University School of Medicine and an investigator in the NEURO-TTRansform study. “Approval of WAINUA represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease.”

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