Remove FDA Approval Remove Gene Remove Gene Expression
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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma.

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Eli Lilly’s Olumiant Wins FDA Approval as First Systemic Treatment for Alopecia Areata

XTalks

Eli Lilly’s Olumiant (baricitinib) has won US Food and Drug Administration (FDA) approval as the first systemic treatment for severe alopecia areata (AA), an autoimmune disorder that leads to patchy baldness. The Olumiant approval is therefore a big win as it helps fulfill a significant unmet need in the area.

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Data dive finds cheap diuretic could be Alzheimer’s drug

pharmaphorum

Bumetanide’s potential was discovered by analysing data on more than 1,300 FDA-approved drugs derived from brain tissue samples, laboratory experiments involving animal and human cells, and human population studies.

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Aardvark Therapeutics IPO Sets Sights on Satiating Hyperphagia and Extreme Hunger

XTalks

More recently, MSN reported on a Cell Genomics study where CRISPR reactivated dormant genes in patient-derived cells, restoring normal gene expression and improving metabolic function an innovation that could address PWS at its genetic roots.

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AI tool may help doctors select best drugs for COVID patients

pharmaphorum

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients.

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Vtama (tapinarof) Cream Gains FDA Approval for the Treatment of Plaque Psoriasis in Adults

XTalks

Tapinarof treatment in a mouse model of psoriasis resulted in the downregulation of inflammatory cytokine expression in skin tissue, which involved IL17A , IL17F , IL19 , IL22 , IL23A and IL1B gene expression. Clinical Trials of Vtama.

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ESMO 2022 – PAOLA-1, ovarian cancer, and treatment innovation

pharmaphorum

It can be caused by different types of mutation, including within the BRCA gene and other forms of altered gene expression. PARP inhibitors, which are now used as a first-line management in newly diagnosed individuals, include the FDA-approved olaparib, as well as rucaparib and niraparib.

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