A review on the rare diseases and subsequent orphan drugs
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022.
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| Idioma: | English |
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Brac University
2024
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| Acesso em linha: | http://hdl.handle.net/10361/24371 |
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10361-243712024-10-22T21:06:03Z A review on the rare diseases and subsequent orphan drugs Oishi, Nuzhat Anjum Azad, Md. Abul Kalam School of Pharmacy, Brac University Duchenne Muscular Dystrophy Transthyretin Amyloid Polyneuropathy Friedreich's ataxia Rare diseases Drug development. Orphan drugs. This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022. Cataloged from PDF version of thesis. Includes bibliographical references (pages 76-109). Worldwide among every existing disease, approximately 5000-8000 are often referred to as rare diseases due to their uncommon, chronic, progressive, degenerative, and life-threatening characteristics. This review specifically deals with Duchenne Muscular Dystrophy, Friedreich's Ataxia, and Transthyretin Amyloid Polyneuropathy; three rare conditions each with the scope of better orphan treatment opportunities. In spite of some orphan drugs’ improving results for treating each rare disease, it has been however recommended that only a precise and effective orphan treatment can provide optimal life. In response to these rare diseases, substantial cooperation among the stakeholders, funders, orphan drug developers, regulatory authorities, policymakers, governments, and immense global effort is crucial. This review paper is hence a nuanced analysis of ongoing strategies to patently identify rare diseases, three specific rare diseases profile along with their impending orphan drug candidates, and their pre-and post-development challenges. Nuzhat Anjum Oishi B. Pharmacy 2024-10-22T06:20:22Z 2024-10-22T06:20:22Z ©2022 2022-06 Thesis ID 15146075 http://hdl.handle.net/10361/24371 en Brac University theses are protected by copyright. They may be viewed from this source for any purpose, but reproduction or distribution in any format is prohibited without written permission. 125 pages application/pdf Brac University |
| institution |
Brac University |
| collection |
Institutional Repository |
| language |
English |
| topic |
Duchenne Muscular Dystrophy Transthyretin Amyloid Polyneuropathy Friedreich's ataxia Rare diseases Drug development. Orphan drugs. |
| spellingShingle |
Duchenne Muscular Dystrophy Transthyretin Amyloid Polyneuropathy Friedreich's ataxia Rare diseases Drug development. Orphan drugs. Oishi, Nuzhat Anjum A review on the rare diseases and subsequent orphan drugs |
| description |
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022. |
| author2 |
Azad, Md. Abul Kalam |
| author_facet |
Azad, Md. Abul Kalam Oishi, Nuzhat Anjum |
| format |
Thesis |
| author |
Oishi, Nuzhat Anjum |
| author_sort |
Oishi, Nuzhat Anjum |
| title |
A review on the rare diseases and subsequent orphan drugs |
| title_short |
A review on the rare diseases and subsequent orphan drugs |
| title_full |
A review on the rare diseases and subsequent orphan drugs |
| title_fullStr |
A review on the rare diseases and subsequent orphan drugs |
| title_full_unstemmed |
A review on the rare diseases and subsequent orphan drugs |
| title_sort |
review on the rare diseases and subsequent orphan drugs |
| publisher |
Brac University |
| publishDate |
2024 |
| url |
http://hdl.handle.net/10361/24371 |
| work_keys_str_mv |
AT oishinuzhatanjum areviewontherarediseasesandsubsequentorphandrugs AT oishinuzhatanjum reviewontherarediseasesandsubsequentorphandrugs |
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1814309821859495936 |