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Rilzabrutinib Sets a New Benchmark in Immune Thrombocytopenia Trials

XTalks

In the trial, platelet response, defined as achieving at least 50,000 platelets per microliter or doubling baseline counts, was seen in 65 percent of patients on rilzabrutinib, compared to 33 percent on placebo. percent platelet response in a study published in The New England Journal of Medicine.

Trials 104
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The Power of Personalization Amid the Changing CRO Landscape

Worldwide Clinical Trials

Amidst a shifting clinical landscape characterized by increasingly complex trial designs and growing patient subpopulations, many contract research organizations (CROs) have adopted a “one-stop-shop” strategic approach. Moreover, these delays impact getting potentially lifesaving therapies to patients in need.

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MASH Clinical Trials: New Approaches and Methodologies

XTalks

The surge in MASH cases stresses the need for early diagnosis, timely intervention and precise, reliable methodologies in clinical trials to evaluate new therapies effectively. Read on to gain insights from Medpace experts about advancements in imaging, histology and innovative techniques for MASH clinical trials.

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Cell and gene therapies: why advanced medicines call for specialised logistics

Pharmaceutical Technology

But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. CRYOPDP’s global teams are acutely aware of the seismic shift in focusing on cell and gene therapy drug development, and we are dedicated to supporting this emerging wave of new cell and gene therapies,” says Rossi.

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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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FDA suggests ways to curb constraints with rare disease gene therapy trials

Pharmaceutical Technology

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases. GlobalData is the parent company of Pharmaceutical Technology.

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Insilico Medicine launches trial for AI-discovered drug

Outsourcing Pharma

The artificial intelligence-driven drug discovery company has dosed its first healthy volunteer in a trial for a candidate to treat idiopathic pulmonary fibrosis.

Trials 141