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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 125
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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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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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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 102
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Results from first randomised controlled trial of genetic counselling for familial and inherited colorectal cancer show significant improvements in patient empowerment

SCIENMAG: Medicine & Health

Glasgow, UK: Genetic counselling is essential when dealing with individuals who are affected by, or at risk of, inherited disease. Although it is known to be useful in helping patients cope with test results and deal with uncertainty, there have been very few randomised controlled trials (RCTs) of its effectiveness.

Trials 82
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What Do Patients Have to Say about Gene Therapy Trials? An Upcoming FDA Public Meeting to Hear from Patients and Caregivers

FDA Law Blog: Drug Discovery

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?