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After surprise trial failure, ALS doctors brace for one less treatment option

BioPharma Drive: Drug Pricing

Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.

Doctors 120
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A smartphone app for the self-management of Perthes’ Disease – a blog for Rare Disease Day 2024

On Medicine

What is Perthes’ disease? Perthes’ Disease is a rare condition that affects the developing femoral head (the “ball” of the “ball and socket” hip joint) of a child due to poor blood supply. This in turn can cause significant pain for children with Perthes’ disease and limit their activity levels significantly.

Disease 109
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Sloane’s 1st ASO treatment

KIF1A

Fortunately, Sloane does not currently have epilepsy, and Megan hopes this treatment will prevent it’s onset as her disease progresses. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication. The post Sloane’s 1st ASO treatment appeared first on KIF1A.

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Novel biosensor could help optimise cancer treatment

Drug Discovery World

This biosensor has the potential to aid the development of new treatments for DNA damage-related diseases by revealing how our bodies repair damaged DNA. The post Novel biosensor could help optimise cancer treatment appeared first on Drug Discovery World (DDW).

Treatment 130
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As ALS research booms, one treatment center finds itself in the spotlight

BioPharma Drive: Drug Pricing

The Healey center is at the front of ALS research and care, earning acclaim from patients, doctors and scientists. Still, the complexities of the disease and of drug development have brought hard-felt losses.

Research 142
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Exploring liver disease therapies  

Drug Target Review

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Why are you focusing on liver disease? As a result, waiting lists continue to grow.

Therapies 101
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The future of lymphoma treatment

Drug Target Review

ARC-02 is our anti-CD79b targeting ADC in development for the treatment of relapsed/refractory (r/r) follicular lymphoma (FL), for which there are currently no approved ADCs, as well as r/r diffuse B-cell lymphoma (DLBCL). The ADC uses the same antibody and drug payload as the approved Polivy®, however, it uses Araris’ linker technology.

Treatment 113