ABOUT hATTR
hATTR causes multiorgan dysfunction due to systemic deposition of amyloid fibrils1,2
hATTR is a protein-misfolding disorder of the transthyretin (TTR) protein1,2
The source of the disease is produced in the liver2,3
Liver
(where 90% of TTR
is produced)
Transthyretin tetramer destabilizes
Tetramer dissociates into monomers
Monomers misfold and aggregate into amyloid fibrils
Amyloid deposits throughout the body
Disease progression is rapid if left untreated4
The stages of progression5,6
Walking without assistance
STAGE 1
Walking with assistance
STAGE 2
Confined to a wheelchair or bed
STAGE 3
LIFE EXPECTANCY IS 5 TO 15 YEARS FROM DIAGNOSIS7
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Explore the disease manifestations of hATTR
See how WAINUA targets TTR at the source
FOOTNOTES
hATTR, hereditary transthyretin-mediated amyloidosis; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis.
REFERENCES
- 1. Nativi-Nicolau JN, Karam C, Khella S, Maurer MS. Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness. Heart Fail Rev. 2022;27(3):785-793.
- 2. Tschöpe C, Elsanhoury A. Treatment of transthyretin amyloid cardiomyopathy: the current options, the future, and the challenges. J Clin Med. 2022;11(8):2148.
- 3. Ioannou A, Fontana M, Gillmore JD. RNA targeting and gene editing strategies for transthyretin amyloidosis. BioDrugs. 2023;37(2):127-142.
- 4. Conceição I, González-Duarte A, Obici L, et al. "Red-flag"-symptom clusters in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2016;21(1):5-9.
- 5. Adams D, Ando Y, Beirão JM, et al. Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy. J Neurol. 2021;268(6):2109-2122.
- 6. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
- 7. Hawkins PN, Ando Y, Dispenzeri A, Gonzalez-Duarte A, Adams D, Suhr OB. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625-638.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
- Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.
ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).
INDICATION
WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
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