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Phase II trial investigates allogeneic CAR-T for large B-cell lymphoma

Drug Discovery World

Allogene Therapeutics and Foresight Diagnostics have announced the initiation of a Phase II trial evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line treatment regimen for newly diagnosed large B-cell lymphoma (LBCL) patients who are likely to relapse.

Trials 147
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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 124
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Patient Perspective: Jamie Troil Goldfarb shares her clinical trial experience [video]

Antidote

For individuals living with a condition like melanoma, volunteering to participate in a clinical trial can be a rewarding experience. Individuals with Jamie’s specific diagnosis are given a 14% 5-year survival rate — but through participating in an immunotherapy clinical trial, Jamie is living without evidence of the disease.

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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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Medicinal cannabis: Why more clinical trials and better access is needed

Drug Discovery World

Big pharma is helping to shape the focus The good news is that clinical trials are on the increase and market education is upping pace. Meanwhile, the Pfizer purchase of Arena Pharmaceuticals, highlights cannabinoid-based bowel disease treatments as a promising area.

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Sickle Cell Disease: The Importance of Amplifying Patient Voices

Advarra

Having a one in four chance of developing sickle cell disease (SCD) is uniquely, and overwhelmingly, familiar to the African American population. Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations.

Disease 98
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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.