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 * Impact on daily living
   
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HOW MANY HAE 
TRADE-OFFS ARE TOO MANY?

Advances in long-term prophylactic treatments for hereditary angioedema (HAE)
have reduced the frequency of attacks and enabled significant advances in
patient care.1 In spite of this, many patients modify their lifestyle and fail
to report mild attacks, with an ongoing impact on daily living.2-8 Does
accepting attacks and daily trade-offs really equate to control?2,5,7,8




PATIENT-FOCUSED CARE IN HAE CONTINUES TO EVOLVE2

 


EXPLORING NEWER TECHNOLOGIES

The launch of C1 esterase inhibitor therapies in 2008 marked a significant
advancement in patient care.1,9 More recently, therapeutic modalities expanded
with novel monoclonal antibodies and small molecule prophylactic therapies.10,11
Now, ongoing discovery continues.2,12



Discover potential new targets




UNCOVERING ONGOING IMPACT

While advances in long-term prophylactic treatments have reduced the frequency
of attacks, patients may still experience an emotional and physical toll from
the disease, including those who rarely have attacks.3,5



Explore the untold stories





STAY INFORMED

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 * REFERENCES

REFERENCES
 1.  Busse PJ, Christiansen SC, Riedl MA, et al. US HAEA Medical Advisory Board
     2020 Guidelines for the Management of Hereditary Angioedema. J Allergy Clin
     Immunol Pract. 2021;9(1):132-150.e3. doi:10.1016/j.jaip.2020.08.046 
 2.  Bork K, Anderson JT, Caballero T, et al. Assessment and management of
     disease burden and quality of life in patients with hereditary angioedema:
     a consensus report. Allergy Asthma Clin Immunol. 2021;17(1):40.
     doi:10.1186/s13223-021-00537-2 
 3.  Jain G, Walter L, Reed C, O'Donnell P, Troy J. How do patients and
     physicians communicate about hereditary angioedema in the United States?.
     PLoS One. 2021;16(12):e0260805. doi:10.1371/journal.pone.0260805 
 4.  US Food and Drug Administration. The Voice of the Patient. May 2018.
     Accessed July 31,
     2024.https://www.fda.gov/files/about%20fda/published/The-Voice-of-the-Patient---Hereditary-Angioedema.pdf 
 5.  Banerji A, Davis KH, Brown TM, et al. Patient-reported burden of hereditary
     angioedema: findings from a patient survey in the United States. Ann Allergy
     Asthma Immunol. 2020;124(6):600-607. doi:10.1016/j.anai.2020.02.018
 6.  Radojicic C, Riedl MA, Craig TJ, et al. Patient perspectives on the
     treatment burden of injectable medication for hereditary angioedema.
     Allergy Asthma Proc. 2021;42(3):S4-S10. doi:10.2500/aap.2021.42.210025
 7.  Mendivil J, Murphy R, de la Cruz M, et al. Clinical characteristics and
     burden of illness in patients with hereditary angioedema: findings from a
     multinational patient survey. Orphanet J Rare Dis. 2021;16(1):94.
     doi:10.1186/s13023-021-01717-4 
 8.  Data on file. Ionis Pharmaceuticals, Inc. Carlsbad, CA. 
 9.  Zuraw BL. HAE therapies: past present and future. Allergy Asthma Clin
     Immunol. 2010;6(1):23. doi:10.1186/1710-1492-6-23 
 10. FDA approves new treatment for rare hereditary disease. Press release. US
     Food and Drug Administration. August 23, 2018. Accessed July 31, 2024.
     https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-treatment-rare-hereditary-disease 
 11. BioCryst Announces FDA Approval of ORLADEYO™ (berotralstat), First Oral,
     Once-daily Therapy to Prevent Attacks in Hereditary Angioedema Patients.
     Press release. BioCryst Pharmaceuticals, Inc. December 3, 2020. Accessed
     July 31, 2024.
     https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-fda-approval-orladeyotm-berotralstat-first 
 12. Covella B, Giliberti M, Montinaro A, Rossi L, Montinaro V. Hereditary
     angioedema: novel molecules for treatment of acute attacks and long-term
     prophylaxis. Future Pharmacol. 2024;4(1):41-53.
     doi:10.3390/futurepharmacol4010005 
 13. Valerieva A, Nedeva D, Yordanova V, Petkova E, Staevska M. Therapeutic
     management of hereditary angioedema: past, present, and future. Balkan Med
     J. 2021;38(2):89-103. doi:10.5152/balkanmedj.2021.21094 
 14. Azmy V, Brooks JP, Hsu FI. Clinical presentation of hereditary angioedema.
     Allergy Asthma Proc. 2020;41(Suppl 1):S18-S21.
     doi:10.2500/aap.2020.41.200065 
 15. TD Cowen Equity Research. Therapeutic Categories Outlook: Orphan
     Diseases/Hereditary Angioedema. Published September
     2023.https://www.cowen.com/insights/?sector=health-care 
 16. López Lera A. Pathophysiology and underlying mechanisms in hereditary
     angioedema. Balkan Med J. 2021;38(2):82-88.
     doi:10.4274/balkanmedj.galenos.2020.2020.10.166 
 17. Collotta D, Bertocchi I, Chiapello E, Collino M. Antisense
     oligonucleotides: a novel frontier in pharmacological strategy. Front
     Pharmacol. 2023;14:1304342. doi:10.3389/fphar.2023.1304342 
 18. Crooke ST, Witztum JL, Bennett CF, Baker BF. RNA-targeted therapeutics.
     Cell Metab. 2018;27(4):714-739. doi:10.1016/j.cmet.2018.03.004. Published
     correction appears in Cell Metab. 2019;29(2):501. doi:
     10.1016/j.cmet.2019.01.001
 19. Riedl MA, Bordone L, Revenko A, Newman KB, Cohn DM. Clinical progress in
     hepatic targeting for novel prophylactic therapies in hereditary
     angioedema. J Allergy Clin Immunol Pract. 2024;12(4):911-918.
     doi:10.1016/j.jaip.2023.12.025
 20. Maurer M, Magerl M, Ansotegui I, et al. The international WAO/EAACI
     guideline for the management of hereditary angioedema—the 2017 revision and
     update. Allergy. 2018;73(8): 1575–1596. doi:10.1111/all.13384


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