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dc.contributor.advisorAzad, Md. Abul Kalam
dc.contributor.authorOishi, Nuzhat Anjum
dc.date.accessioned2024-10-22T06:20:22Z
dc.date.available2024-10-22T06:20:22Z
dc.date.copyright©2022
dc.date.issued2022-06
dc.identifier.otherID 15146075
dc.identifier.urihttp://hdl.handle.net/10361/24371
dc.descriptionThis thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022.en_US
dc.descriptionCataloged from PDF version of thesis.
dc.descriptionIncludes bibliographical references (pages 76-109).
dc.description.abstractWorldwide 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.en_US
dc.description.statementofresponsibilityNuzhat Anjum Oishi
dc.format.extent125 pages
dc.language.isoenen_US
dc.publisherBrac Universityen_US
dc.rightsBrac 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.
dc.subjectDuchenne Muscular Dystrophyen_US
dc.subjectTransthyretin Amyloid Polyneuropathyen_US
dc.subjectFriedreich's ataxiaen_US
dc.subjectRare diseasesen_US
dc.subject.lcshDrug development.
dc.subject.lcshOrphan drugs.
dc.titleA review on the rare diseases and subsequent orphan drugsen_US
dc.typeThesisen_US
dc.contributor.departmentSchool of Pharmacy, Brac University
dc.description.degreeB. Pharmacy


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