Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis
Hereditary angioedema (HAE) is a rare disease caused by a genetic alteration of the <i>SERPING1</i> gene and characterized by recurrent attacks of angioedema that involve the skin, and the mucosae of the gastrointestinal tract and upper airways, which significantly affect the quality of...
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| Format: | Article |
| Language: | English |
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MDPI AG
2024-01-01
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| Series: | Future Pharmacology |
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| Online Access: | https://www.mdpi.com/2673-9879/4/1/5 |
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| author | Bianca Covella Marica Giliberti Adriano Montinaro Luigi Rossi Vincenzo Montinaro |
| author_facet | Bianca Covella Marica Giliberti Adriano Montinaro Luigi Rossi Vincenzo Montinaro |
| author_sort | Bianca Covella |
| collection | DOAJ |
| description | Hereditary angioedema (HAE) is a rare disease caused by a genetic alteration of the <i>SERPING1</i> gene and characterized by recurrent attacks of angioedema that involve the skin, and the mucosae of the gastrointestinal tract and upper airways, which significantly affect the quality of life of patients. Nowadays there are effective drugs for both 1. treating acute attacks and 2. preventing attacks with a long-term prophylaxis. However, there are some unmet needs for HAE treatment, and therefore several novel molecules are under active testing for this clinical condition. Novel drugs will simplify the mode of administration (oral versus parenteral for both on demand treatment or long-term prophylaxis), prolong the interval between administrations (up to 3–6 months of efficacy with a single administration), target more specifically the central enzymes involved in the generation of bradykinin, the ultimate mediator of angioedema (prekallikrein, activated plasma kallikrein or activated factor XII), and potentially determine a definitive cure for the disease by genetic manipulation of the altered gene (<i>SERPING1</i>) or other downstream genes (<i>KLKB1</i>). In this review we provide a panoramic view of all new medications that are under active experimentation and will probably transform and enrich all of the therapeutic armamentarium for treating this disease. |
| first_indexed | 2024-04-24T18:15:27Z |
| format | Article |
| id | doaj.art-1855289692fd42d092be00d17527c654 |
| institution | Directory Open Access Journal |
| issn | 2673-9879 |
| language | English |
| last_indexed | 2024-04-24T18:15:27Z |
| publishDate | 2024-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Future Pharmacology |
| spelling | doaj.art-1855289692fd42d092be00d17527c6542024-03-27T13:42:20ZengMDPI AGFuture Pharmacology2673-98792024-01-0141415310.3390/futurepharmacol4010005Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term ProphylaxisBianca Covella0Marica Giliberti1Adriano Montinaro2Luigi Rossi3Vincenzo Montinaro4Division of Nephrology, “Miulli” General Regional Hospital, 70021 Acquaviva delle Fonti, BA, ItalyDivision of Nephrology, Azienda Ospedaliero-Universitaria Policlinico, 70124 Bari, BA, ItalyDivision of Nephrology, Azienda Ospedaliero-Universitaria Policlinico, 70124 Bari, BA, ItalyDivision of Nephrology, “Miulli” General Regional Hospital, 70021 Acquaviva delle Fonti, BA, ItalyDivision of Nephrology, “Miulli” General Regional Hospital, 70021 Acquaviva delle Fonti, BA, ItalyHereditary angioedema (HAE) is a rare disease caused by a genetic alteration of the <i>SERPING1</i> gene and characterized by recurrent attacks of angioedema that involve the skin, and the mucosae of the gastrointestinal tract and upper airways, which significantly affect the quality of life of patients. Nowadays there are effective drugs for both 1. treating acute attacks and 2. preventing attacks with a long-term prophylaxis. However, there are some unmet needs for HAE treatment, and therefore several novel molecules are under active testing for this clinical condition. Novel drugs will simplify the mode of administration (oral versus parenteral for both on demand treatment or long-term prophylaxis), prolong the interval between administrations (up to 3–6 months of efficacy with a single administration), target more specifically the central enzymes involved in the generation of bradykinin, the ultimate mediator of angioedema (prekallikrein, activated plasma kallikrein or activated factor XII), and potentially determine a definitive cure for the disease by genetic manipulation of the altered gene (<i>SERPING1</i>) or other downstream genes (<i>KLKB1</i>). In this review we provide a panoramic view of all new medications that are under active experimentation and will probably transform and enrich all of the therapeutic armamentarium for treating this disease.https://www.mdpi.com/2673-9879/4/1/5angioedemahereditaryC1 inhibitorpharmacologicaltreatmentbradykinin |
| spellingShingle | Bianca Covella Marica Giliberti Adriano Montinaro Luigi Rossi Vincenzo Montinaro Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis Future Pharmacology angioedema hereditary C1 inhibitor pharmacological treatment bradykinin |
| title | Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis |
| title_full | Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis |
| title_fullStr | Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis |
| title_full_unstemmed | Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis |
| title_short | Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis |
| title_sort | hereditary angioedema novel molecules for treatment of acute attacks and long term prophylaxis |
| topic | angioedema hereditary C1 inhibitor pharmacological treatment bradykinin |
| url | https://www.mdpi.com/2673-9879/4/1/5 |
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