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MHRA approves first gene therapy for infusion into the brain

European Pharmaceutical Review

The product, produced by biopharma company PTC Therapeutics, is approved for patients 18 months and over. Upstaza is administered through a minimally invasive neurosurgical stereotactic procedure used to treat several neurological disorders in paediatric and adult patients. Upstaza clinical trials.

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Liver injuries prompt clinical hold on Sanofi BTK drug

pharmaphorum

billion buyout of Principia Biopharma has run into trouble, after the FDA placed it on partial clinical hold while a safety signal is investigated. Those changes haven’t been enough to prevent the FDA from taking action, although outside the US the studies will continue as planned with the tighter safety monitoring.

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Newron says pivotal treatment-resistant schizophrenia trial is a go

pharmaphorum

The Italian biopharma said it had decided to press ahead with the new study after assessing results from 100 patients with TRS in open-label studies who received the glutamate modulator drug as an add-on to their current therapy for more than six months. in the evenamide group.

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Leading speciality pharmacies in pharma

Pharmaceutical Technology

Speciality pharmacies play a crucial role in medication dosing, disease and side effects management, and patient care. The expert services rendered by them help in improving patient outcomes, minimising costs of the healthcare system, improving medicinal adherence and persistency and saving clinicians’ time.

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FDA slaps partial hold on Biogen, InnoCare MS drug

pharmaphorum

The US regulator has placed oral BTK inhibitor orelabrutinib on partial clinical hold, meaning new patients cannot be enrolled into clinical trials and patients treated with the drug for 70 days or less must discontinue treatment. billion buyout of Principia Biopharma.

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How to Work with Patient Advocates, Influencers, and Ambassadors

PM360

Only 5% of patients ranked pharma companies as the most trusted player in the healthcare space, according to a 2021 survey of 500 patients from Accenture. But 71% also want publicly shared information about how patients’ perspectives were incorporated into the development of products and services. Noël Moossa.

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The evolution of AAVs in cell and gene therapy

European Pharmaceutical Review

Dr Baghirzade continued, highlighting that since a particular defining moment for the industry in the late 90s, which resulted in an unfortunate patient fatality, “there is a lot more focus on safety” in gene therapy clinical trials today. She also touched on production costs being irrecoverable—there is a high cost per patient.