This unfortunate news is far from surprising. The problem is two-fold: minimal dystrophin expression and failure to account for accumulated muscle damage. Combinatorial treatment with AVGN7 could address the latter and possibly improve the former. Interested companies should contact AAVogen. https://lnkd.in/eFabEFZQ
AAVogen’s Post
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In patients with various forms of transthyretin amyloidosis, including transthyretin amyloid polyneuropathy, a reliable, useful tool for investigating the disease and how to rank drug candidates is X-ray crystallography, according to findings from an analysis published in the journal Molecules MDPI. Read more: https://brnw.ch/21wHO69 #RareDisease
X-Ray Crystallography Useful to Research Treatments for ATTR-PN
https://www.rarediseaseadvisor.com
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Really excited to see this progress in a very classic but complex pathophenotype of MS. Lots to consider, but am very curious about biomarker plans once in MS subjects - could it be MTR/MTSat or maybe myelin water and multi shell diffusion in PRL/NonPRL lesions and what will the remyelinating compounds be combined with? #myelin #multiplesclerosis https://lnkd.in/eZUgFb-w
ACTRIMS 2024: 2 treatments to aid remyelination in MS nearing...
https://multiplesclerosisnewstoday.com
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Understanding the roles of individual conformational states in signaling efficacy and bias will help us to design drugs that discriminately target a disease-related conformation. My first BioRxiv manuscript is online, which focuses on determining the structure of the intermediate GPCR-G protein complex and its function in signaling. Guided by a conformational landscape profiled by 19F quantitative NMR (19F-qNMR) and MD, we resolved the first structure of an unliganded GPCR-Gαsβγ intermediate complex with a resolution of 2.8A by a conformational blockade strategy to capture the transient state. More importantly. we presented direct evidence that the intermediate GPCR-G protein complex initiates a rate-limited nucleotide exchange without progressing to the fully activated end-state complex, thereby bridging a critical gap in our understanding the complexity of GPCR signaling, including efficacy and selectivity. https://lnkd.in/eVpM5uaS
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New paper alert! 💥 I just had a paper published on Viltolarsen, the newest medication for Duchenne's muscular dystrophy, and there's been a lot of controversy over its approval. Check out my thoughts on this new drug. #DuchennesMuscularDystrophy #NewDrug #Controversy #MedicalResearch #RegisUniversity #viltolarsen
Viltolarsen: a treatment option for Duchenne muscular dystrophy patients who are amenable to exon 53 skipping therapy
tandfonline.com
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A good day to share the publication of the validation of FoundationOne Tracker, a therapy response ctDNA monitoring assay. https://lnkd.in/ecdQHDBQ
Analytical validation of a novel comprehensive genomic profiling informed circulating tumor DNA monitoring assay for solid tumors
journals.plos.org
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A novel form of conditional CAR-T armouring with IL-18 that only releases the cytokine when CAR-T cells are activated has been published by John Maher and colleagues. This is an elegant solution to the toxicity of constitutive IL-18 release, yet retains constitutive production of IL-18 by substituting the caspase 1 cleavage site within pro-IL18 with that preferred by granzyme B, yielding GzB-IL18. Only when the CAR is active and granzyme is released does the IL-18 become liberated. https://lnkd.in/enZ374BV
Granzyme B-activated IL18 potentiates αβ and γδ CAR T cell immunotherapy in a tumor-dependent manner
cell.com
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Excited to announce our recent publication, highlighting the essential role of ANGPTL8 in insulin signaling and differentiation in pre-adipocytes. Explore the details at https://lnkd.in/eU5MXTAh
Silencing ANGPTL8 reduces mouse preadipocyte differentiation and insulin signaling
sciencedirect.com
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Sequential, multistep activation mechanism of metabotropic glutamate receptor subtype 5. Just out. https://lnkd.in/dCaR7J96
Stepwise activation of a metabotropic glutamate receptor - Nature
nature.com
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Ligand efficacy modulates conformational dynamics of the µ-opioid receptor Nature https://lnkd.in/enqpAXkj
Ligand efficacy modulates conformational dynamics of the µ-opioid receptor - Nature
nature.com
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👉 "Findings highlight the promising potential of artemisinins as effective drugs for the comprehensive treatment of PCOS." https://lnkd.in/e4pzrF5H 🔗 Data demonstrated the efficacy of artemisinins in alleviating symptoms associated w/ PCOS in both rodent models & human patients 👉 "Artemisinins, compounds known for their antimalarial properties, suppress ovarian androgen synthesis by promoting degradation of cytochrome P450 11A1 (CYP11A1), making them a promising new approach for preventing and treating PCOS." "Remedy hope for polycystic ovary syndrome", by Prof Elisabet Stener-Victorin 🔗 https://lnkd.in/eX_eUMs2
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