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For rare disease patients in Mainland China, hope of greater access to treatment

Clarivate

Recent policy and regulatory moves have begun to sketch out a framework for rare disease drug approvals in the country, but obstacles to approvals and patient access remain. Mainland China has taken significant steps toward improving access to treatment for its estimated 20 million rare disease patients in recent years. [1]

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Leading in Pharma: a woman’s pathway

European Pharmaceutical Review

After moving to London in 2011, Ekaterina worked in global health and medical philanthropy, focusing on improving outcomes in maternal and child health. I used to prescribe antidepressants for patients but never considered psychiatry as a profession.

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Using RWE in rare disease drug development: effective innovations with historical controls

European Pharmaceutical Review

Rare disease drug development poses unique challenges that can be overcome by using real-world evidence (RWE). Small populations, paediatric patients, and the desire from patients and their caregivers to receive active therapy can make the conduct of randomised trials with placebo control untenable.

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Paving the way for anti-Abeta active immunotherapy

European Pharmaceutical Review

Targeting toxic species of Abeta, such as multimers (with aducanumab and lecanemab) or pyroglutamate Abeta3-42 (with donanemab), induced significant removal of Abeta plaque load in the brains of Alzheimer’s patients, leading to the delay in cognitive decline. Treatment with the mAbs delayed cognitive decline by 2.6 months for lecanemab and 5.5

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A history of blood cancer treatment

pharmaphorum

Over the past two centuries, researchers have identified more than 100 different types of blood cancer, while most patients may be familiar with the big three (leukaemia, lymphoma, and melanoma). In 39-year-old patient Sarah Newbury, Solly observed the appearance of fatigue and bone pain resulting from multiple fractures.

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Three biggest threats to healthcare

World of DTC Marketing

Right now, voters are worried about inflation and rising fuel prices, but they ignore the imminent threats to their healthcare. American healthcare continues to be under assault, and unless we address these issues, a severe health issue could bankrupt families. THREAT 3: RISING HEALTHCARE COSTS. THREAT 1: OBESITY AND DIABETES.