Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. Cell and gene therapies are not your typical clinical supply chain.

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The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. Safety is critical in all clinical trials, but with ultra-rare diseases, even greater care is taken. they can help many patients.”.

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Drug Discovery World

JULY 11, 2022

These challenges exist across the translation cycle from early research to clinical trials, manufacturing, and ultimately reimbursement. The complexities of sample management can significantly extend the timelines for clinical trials. Methods& clinical development, 21,524–529. Regulatory requirements.

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The Pharma Data

DECEMBER 8, 2020

The impact of CNS diseases extends beyond patients—to their families and society as well.” The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . Most read today on BioSpace: Source link.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies.

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