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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. The most common side effects observed were initial insomnia, irritability and dyskinesia. The efficacy and safety profile of Upstaza has been shown across clinical trials and compassionate use programmes.

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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. .

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$5 billion bispecific antibody pharma collaboration

European Pharmaceutical Review

Biopharma Akeso Inc. state the therapy could help reduce side effects and safety concerns. has announced it will enter into a collaboration potentially worth up to $5 billion with Summit Therapeutics Inc., Under the agreement, Akeso Inc. will receive a $500 million upfront payment. A $5 billion collaboration.

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Practical advice for Early Scientific Advice (ESA) in HTA submissions

pharmaphorum

In recent years, the biopharma market has become progressively complex. Payers must take into consideration not only the clinical efficacy of a drug, but also its safety profile, side effects, and cost-effectiveness, among other factors.

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FDA slaps partial hold on Biogen, InnoCare MS drug

pharmaphorum

billion buyout of Principia Biopharma. That could give the drugs an advantage over CD20-targeting therapies like Roche’s Ocrevus (ocrelizumab) that inhibit B cells across the board, leaving patients prone to infections and other side effects.

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AUTHENTIC PATIENT VOICE: BOT TO TROT AI Steps Up to Transform Healthcare

PM360

As we enter into the age of precision medicine, AI will be a critical tool to determine, with the help of disease knowledge graphs, which indications, associations, contraindications, side effects, genetic markers, drug targets, drug carriers, and interactions to take into account on a case-by-case basis. AI isn’t coming for your job.