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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


“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.”

Presenting at the Amyloidosis Support Groups bi-annual ATTR conference

Hosted by Muriel Finkel and the Amyloidosis Support Groups (ASG), Deb Boedicker presented the Amyloidosis Speakers Bureau at the ASG bi-annual ATTR conference.

Mackenzie’s Mission joins the BU School of Medicine Dean’s Advisory Board

Mackenzie’s Mission is proud to join the Boston University Chobanian & Avedisian School of Medicine Dean’s Advisory Board (DAB) as a donor representative of the Amyloidosis Center, and nominated by Dr. Vaishali Sanchorawala.

The DAB provides the dean with counsel on issues affecting the school including strategic planning, external relations, and fundraising initiatives that facilitate the achievement of the dean’s vision for the School of Medicine.

Given our strong commitment to medical education, we look forward to supporting the dean (Dr. Karen Antman) and her important goals for future physicians.

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