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A Complex Chromosomal Abberation Called Adrenoleukodystrophy

Delveinsight

Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. A novel, brain penetrant, orally bioavailable and selective PPAR?

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Considerations for Neuroscience Trials with Intrathecal Delivery and Other Methods of Direct CNS Administration

XTalks

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. What About Small Proteins and Antibodies Reaching Extracellular CNS Targets? Viral Vectors for Gene Delivery to the CNS.

Trials 83
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How Can Broadening Our Knowledge in Bioconjugate Design and Development Help Develop Better Medicines to Treat Oncology and Beyond?

XTalks

Dr. Dieterich elaborated on the critical aspects of each component: “There’s the protein part, a small molecule and then there’s the linker part in the middle.” These changes in the tumor environment help the tumor resist treatment by modifying gene activity, avoiding immune detection and supporting survival pathways.

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Nanoparticle Formulation: A Paradigm Shift towards Novel Drug Delivery

Roots Analysis

Owing to their unique size and physicochemical properties ( surface roughness, surface area, surface energy, crystal structure and shape ), nanoparticles can be widely used as a contrasting agent in medical imaging, a vesicle to cross the blood-brain barrier and a carrier for targeted delivery of genes / drugs, proteins, vaccines and antibiotics.

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Biopharmaceutical Excipient Manufacturing – Current Scenario and Future Trends

Roots Analysis

In the last decade alone, the annual number of approvals of biopharmaceuticals (including monoclonal antibodies, recombinant proteins, vaccines and gene therapies), by the US FDA, have steadily risen. Over the years, the rising popularity of biologics has led to a paradigm shift in the healthcare industry.

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The Power of Lipids: Enhancing Drug Delivery with Nanoparticles

Roots Analysis

In fact, it has been observed that around 40% of the pharmaceutical drugs approved by regulatory organizations exhibit poor bioavailability / solubility. Further, every year, poor bioavailability is one of the main causes of drug failure in obtaining approval authorization.