Publications by authors named "Laura Sayegh"

Background: Hereditary angioedema (HAE) is a rare genetic disease characterized by recurrent episodes of cutaneous or subcutaneous edema. There is clinical need for treatments that reduce the rate of HAE attacks in patients.

Objectives: Primary objectives were to evaluate the effectiveness of lanadelumab on attack free rate (AFR; proportion of patients who had zero HAE attacks), and on every two weeks (Q2W) and every four weeks (Q4W) adjustments on AFR.

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Article Synopsis
  • - Natural history studies in rare diseases often seek to identify genetic mutations to help develop targeted treatments, but there is uncertainty about how these studies are regulated globally.
  • - A review of regulations across 19 countries found varied classifications of natural history studies, with 37% seen as non-interventional; many countries lack clear guidelines on genetic testing and genetic counseling requirements.
  • - The inconsistent regulatory landscape complicates multinational research in orphan diseases, highlighting the need for standardized international guidelines, which may improve efficiency as new ICH guidelines emerge.
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Background: Rapidly disintegrating or 'fast-melt' oral formulations have been developed recently to facilitate drug intake among patients. Even though these formulations have helped to improve therapy adherence, some of their limitations include: the dissolution time, their facility to be swallowed, and the dosage strengths that may be accommodated. To overcome these limitations, a novel, porous, quickly disintegrating, and easier-to-swallow fast-melt formulation based on powder-liquid, three-dimensional printing (3DP) technology has been developed.

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Acetylcholine (ACh) is a neurotransmitter/neuromodulator in the nematode nervous system and induces its effects through interaction with both ligand-gated ion channels (LGICs) and G protein-coupled receptors (GPCRs). The structure, pharmacology and physiological importance of LGICs have been appreciably elucidated in model nematodes, including parasitic species where they are targets for anthelmintic drugs. Significantly less, however, is understood about nematode ACh GPCRs, termed GARs (G protein-linked ACh receptors).

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