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Lumos Pharma’s Patient-Centric Strategy for Rare Disease Drug Development

XTalks

Among these, rare pediatric diseases present unique challenges and opportunities for rare disease drug development. Pediatric growth hormone deficiency (PGHD) is one such condition, affecting a child’s growth and development. This venture also led me to identify a gap in the market for contract manufacturing of recombinant proteins.

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Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder

XTalks

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

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Thirteen potential blockbuster drugs to watch in 2024

Drug Discovery World

Thirteen new-to-market therapeutics and drugs poised to launch in 2024 will achieve ‘blockbuster’ status by 2029 or deliver game-changing benefits to patients. The 2024 Drugs to Watch, are: 1. Due to a delayed US launch it remains a drug to watch for 2024.

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Outsourcing Peptide Synthesis: Preferred Choice of Drug Developers

Roots Analysis

Presently, over 80 peptide-based drugs are available in the market for myriad of disorders and diseases. The growing popularity of peptide-based drugs can be attributed to its high specificity, minimal drug-drug interactions, affinity, proven pharmacological value and favorable safety profiles.

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Efruxifermin Combined with GLP-1RA Shows Clinical Efficacy in MASH, Study Reveals

XTalks

Efruxifermin is a novel Fc-FGF21 fusion protein designed to emulate the activity of native FGF21, an endogenous hormone that regulates metabolism and alleviates cellular stress. No serious adverse events related to the drug were reported. MASH is intricately linked to cardiovascular, kidney and metabolic disorders.

Insulin 52
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COVID-19 and Melatonin: Researchers Use AI To Study New Treatment Option

XTalks

In addition to innovative new antibody drugs and mRNA vaccines, researchers have found that the sleep-regulating hormone melatonin may also offer protection against COVID-19. Lerner researchers developed a novel artificial intelligence platform to identify potential drugs for COVID-19, finding melatonin to be a promising candidate.

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World Lupus Day 2023 and Novel Investigational Drugs for Lupus

XTalks

The exact cause of lupus is not entirely understood, but it is thought to be from a combination of genetic, environmental and hormonal factors. GSK’s Benlysta (belimumab) is a monoclonal antibody that was approved by the US Food and Drug Administration (FDA) in 2011 for the treatment of SLE.

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