Remove tag long-term-treatment
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Cost of disease-modifying therapies for multiple sclerosis continues to rise in US

Pharmaceutical Technology

Currently, most patients face the prospect of life-long treatment with MS medications, as the main objectives of available disease-modifying therapies (DMTs) are to reduce the frequency of relapses and to delay the accumulation of disability. DMTs for MS have a high price tag, particularly in the US.

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Free access to Zolgensma curbed, says Novartis

pharmaphorum

Since 2020, the global SMA treatment landscape has progressed, with three treatments now approved, according to Novartis. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

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A broad range of unmet needs remains in the immuno-oncology space

Pharmaceutical Technology

Immuno-oncology (IO) agents have transformed the cancer therapeutics landscape, driving long-term remissions in a subset of patients who historically had limited options. Efficacy-related unmet needs included the need for a greater long-term benefit, higher response rates, and mechanisms for overcoming resistance to IO therapies.

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Orphan drugs’ financial success raises questions

pharmaphorum

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. As covered in a previous article , the importance of R&D to develop treatments for rare diseases is high. It is estimated that between 3.5 The price is right.

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Advanced therapies and the high-profile pricing dilemma

pharmaphorum

The advancements made in drug development over the last decade have seen the arrival of a number of treatments that offer one-time cures and more effective therapies for conditions in smaller patient populations. Advanced therapies have changed the treatment landscape for the industry and for patients. Changing the model.

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Realising milestones with gene therapy for SMA

European Pharmaceutical Review

Novartis shared long-term data for its SMA gene therapy in March 2023, which indicated significant long-term motor-milestone achievements in spinal muscular atrophy (SMA). There are three modes of action (MOA) available for treatments – intrathecal injection, intravenous infusion and an oral formula.

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bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

pharmaphorum

There is a curative treatment for people who rely on blood transfusions to survive and maintain their levels of red blood cells. In this first draft guidance NICE raised a series of issues with Zynteglo, which bluebird has already agreed to supply at a confidential discount from its hefty price tag, which is around €1.57

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