Kenneth B. Margulies

Dec 24, 2024 | mdpi.com | Kenneth B. Margulies

4. DiscussionThere are essential evaluations central to the IND application comprising details on the investigative product (IP) manufacturing process, systemic toxicity evaluation, and IP disposition (ADME/PK) properties. Due to the dispositional nature of protein therapeutics, the ADME properties are less relevant when compared to small-molecule therapeutics.

Sep 17, 2024 | nature.com | Khanh V. Doan |Won Dong Lee |Sarmistha Mukherjee |Gabriel K. Adzika |Ryan B. Gaspar |Corey Holman | +3 more

AbstractNicotinamide adenine dinucleotide (NAD+) is an essential co-factor in metabolic reactions and co-substrate for signaling enzymes. Failing human hearts display decreased expression of the major NAD+ biosynthetic enzyme nicotinamide phosphoribosyltransferase (Nampt) and lower NAD+ levels, and supplementation with NAD+ precursors is protective in preclinical models.

Jul 10, 2023 | pubmed.ncbi.nlm.nih.gov | Lee BW |Benjamin Lee |Kenneth B. Margulies |Margulies KB

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Jun 1, 2023 | jacc.org | Benjamin Lee |Kenneth B. Margulies

JACC Journals › JACC: BTS › Archives › Vol. 8 No. 6 IntroductionDuchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), together called the dystrophinopathies, are a group of X-linked recessive disorders leading to weakness, muscle wasting, and premature death.