Mon.May 23, 2022

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PTC gets green light for gene therapy Upstaza in EU

pharmaphorum

PTC Therapeutic’s Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects for gene therapies in the bloc. Once full approval is granted Upstaza (eladocagene exuparvovec) will become the first disease-modifying treatment for AADC (aromatic L-amino acid decarboxylase) deficiency, as well as the first gene therapy directly administered into the brain, said PTC.

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PTC Therapeutic’s Upstaza has been recommended by both the EMA and CHMP

Pharma Times

It is the first gene therapy to be directly administered into the brain to treat aromatic L-amino acid decarboxylase (AADC) deficiency

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Takeda, Daiichi-backed wearable study planned in Japan

pharmaphorum

A study that will look at the feasibility of tracking people’s lifestyle habits for an extended period using a wearable device has got underway in Japan, backed by drugmakers Takeda and Daiichi Sankyo. The device will be worn by 2,000 people for a year, harvesting data on sleep status, heart rate, and activity levels, according to the study partners, which also include digital health and telemedicine firm MICIN and Tohoku University.

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Bharti Rai, Novartis VP & Head of Commercial Acceleration on What’s Ahead for 1st and 3rd party data, Insights, Analytics & Digital Data

Pharma Marketing Network

Tune in to this episode of the Pharma Marketing Podcast with Bharti Rai, Novartis VP, Chief Commercial Operations, Insight, Analytics Officer Data & Digital, for a deeper dive into what’s ahead for 1st and 3rd party data, insights, analytics & digital data. The post Bharti Rai, Novartis VP & Head of Commercial Acceleration on What’s Ahead for 1st and 3rd party data, Insights, Analytics & Digital Data appeared first on Pharma Marketing Network.

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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

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Amwell’s SilverCloud buy yields new digital tool for mental health

pharmaphorum

Telehealth company Amwell has marked Mental Health Awareness month with the launch of a digital tool that will deliver cognitive behavioural health interventions across a spectrum of mental illness. The suite – which combines virtual therapy and psychiatry, optional coaching, and digital mental health programmes tailored to the patient’s needs – has been developed in the wake of Amwell’s acquisition last year of SilverCloud Health.

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Dermatology drug developer taps into Veeva engagement tool

Outsourcing Pharma

With Veeva Link for Key People, LEO Pharma is looking to deepen its efforts to engage with knowledgeable medical professionals in the dermatology community.

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More Trending

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CHMP recommends Sanofi’s Xenpozyme for rare disease ASMD

Pharma Times

Positive opinion is based on data from clinical trials which showed improvement in lung function

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Dupixent claims new territory in eosinophilic oesophagitis

pharmaphorum

Sanofi and Regeneron have added another string to the bow of their immunology blockbuster Dupixent with an FDA approval in eosinophilic oesophagitis (EoE), which could help the drug reach its €10 billion peak sales target. Dupixent (dupilumab) made more than €5 billion last year from its current uses in atopic dermatitis, eosinophilic asthma and chronic rhinosinusitis with nasal polyps, and EoE is one of a clutch of additional indications that Sanofi and Regeneron are relying on to push sales ev

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Tips for selecting a construction partner

Pharma Manufacturing

For pharma, picking the right partner can have an enormous impact on project outcomes

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USAID on expanding digital health globally – part two

pharmaphorum

This article follows part one of our two-part series, in which we discussed how USAID invests in digital infrastructure as part of its development and humanitarian assistance programmes. . Adele Waugaman, senior digital health coordinator of the COVID-19 response at the United States Agency for International Development (USAID), discusses the value of establishing donor partnerships and how those collaborations can improve digital health options globally. .

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Position Your Pharmacy for Expansion

Speaker: Chris Antypas and Josh Halladay

Access to limited distribution drugs and payer contracts are key to pharmacy expansion. But how do you prepare your operations to take the next step? Meaningful data: Collect and share clinical data regarding outcomes, utilization, and more Reporting: Limited distribution models require efficient tracking and reporting systems Workflows: Align workflows with specific pharma and payer contractual requirements For in-depth, expert insights on pharmacy expansion, watch this webinar from Inovalon.

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[Solutions Spotlight] Stop the stall: The time is now for predictive maintenance in pharma

Pharma Manufacturing

Andrea Corona is joined by Zach Gilulah, pharma machine health lead at Augury.

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CHMP backs Sanofi’s Xenpozyme as first drug for ASMD

pharmaphorum

Sanofi’s Genzyme unit has moved a step closer to securing EU approval for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after scoring a positive opinion from the CHMP. Xenpozyme (olipudase alfa) is set to become the first and only approved therapy for ASMD type A/B or ASMD type B, two forms of a group of genetic disorders also known as Niemann-Pick disease in which fats build up within cells, causing them to die and disrupting tissue

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