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Genetic disease

This page shows the latest Genetic disease news and features for those working in and with pharma, biotech and healthcare.

Biogen's ALS drug tofersen misses phase 3 endpoint

Biogen's ALS drug tofersen misses phase 3 endpoint

ALS). ALS is a progressive, fatal neurodegenerative disease with an average survival of 3-5 years and SOD1-ALS is a rare, genetic form that accounts for 2% of the estimated ... pre-symptomatic individuals with a SOD1 genetic mutation and biomarker

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Latest Intelligence

  • EU cross-border healthcare EU cross-border healthcare

    Time can be a precious luxury to someone diagnosed with a rare genetic disease, so getting timely access to new innovative therapies is critical. ... In the last few years, the promise of gene and cell therapies has finally become tangible for certain

  • Rare genetic diseases – what’s in store for 2021? Rare genetic diseases – what’s in store for 2021?

    In 2021, I expect there to be an increase in digital health solutions on the whole but particularly in rare genetic disease areas. ... In. rare genetic disease research, there may be a way to apply Bayesian models to analyse historic data to accelerate

  • Biopharma and orphan drugs Biopharma and orphan drugs

    Such support, coupled with new approaches to patient recruitment and outreach have made rare disease R&D much more common. ... Price concerns. Another argument in favour of the support given to the orphan drug industry is that rare disease drugs generate

  • It started with a miss It started with a miss

    Similarly, commercial teams need to drill down and understand HTA requirements within the disease category to ensure trials are designed to capture the necessary data for modelling. ... In today’s marketplace, with the level of innovation coming

  • The long and winding road to asthma breakthroughs The long and winding road to asthma breakthroughs

    Nucala, which has the market boost of being licensed for home administration via self- administered auto-injector and prefilled syringe, is running smoothly with approval for the rare disease EGPA in ... One of the strengths of GSK is that we are keen to

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Latest appointments

  • New hires at Crescendo, Healx and Galecto New hires at Crescendo, Healx and Galecto

    He co-founded the rare disease patient group support charity Findacure with Nick Sireau, the advisor to and the inspiration behind Healx. ... Hall’s experience in drug development includes significant contributions to the repurposing of nitisinone for

  • BMS appoints new commercial leader, Davies joins Eisai and more BMS appoints new commercial leader, Davies joins Eisai and more

    The field of genomic medicine has the potential to revolutionise our approach to rare genetic disease diagnosis and management, as well as in other areas of personalised medicine.”. ... Sobi appoints head of technical operations. Rare disease

  • Abeona Therapeutics names CEO Abeona Therapeutics names CEO

    Carsten Thiel joins the biopharma from Alexion. Rare genetic disease focused biopharma Abeona Therapeutics has named its chief executive officer in the form of Carsten Thiel. ... Steven Rouhandeh, executive chairman of Abeona, said: “As CEO, Carsten

  • Agios appoints Celgene’s Jackie Fouse Agios appoints Celgene’s Jackie Fouse

    She joins the pharma group’s board of directors. Cancer and rare genetic disease focused group Agios Pharmaceuticals has appointed Jackie Fouse to its board of directors as the company prepares

  • Jonathan Fox leaves AstraZeneca for MyoKardia Jonathan Fox leaves AstraZeneca for MyoKardia

    Will lead programme to target genetic heart disease. MyoKardia has appointed former VP, clinical development at AstraZeneca Jonathan Fox to chief medical officer. ... Commenting on his appointment, he highlighted MyoKardia's potential in treating genetic

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Latest from PMHub

  • Porphyria Awareness Week – Round Up

    The cause of this genetic disease involves a problem with the production of heme, a component in haemoglobin. ... Our medical writing and creative teams collaborated to create an  Infographic to better explain the disease. .

  • Rare Disease Day in a COVID-19 environment

    1). With 8 out of 10 rare diseases considered to have a genetic component. ... My role can begin…) If a couple are planning on becoming pregnant and there is a rare disease in the family that’s genetic, if we find something on the scan

  • #DemandDiversity: The science behind diversity #DemandDiversity: The science behind diversity

    2) A 2018 analysis of studies looking for genetic variants associated with disease found that 78% of study participants were of European ancestry, compared with 10% of Asian ancestry and 2% ... from thousands of participants to find genetic variants

  • October 2020: diversity and inclusion in clinical trials round-up October 2020: diversity and inclusion in clinical trials round-up

    Disparities in sickle cell disease. Globally, sickle cell disease is among the most common genetic disorders and predominantly affects individuals from African, Mediterranean, Arabian, and Indian origins. ... In such settings, specialised sickle cell

  • Reflections of a Fireside Chat from the World Orphan Drug Congress 2020 Reflections of a Fireside Chat from the World Orphan Drug Congress 2020

    rare genetic disease diagnoses, such as Severe Combined Immune Deficiency (SKID) and Metachromatic leukodystrophy (MLD), are different. ... The face of the disease is, thanks to Dr Gaspar and his colleagues’ work, now permanently changing for the better

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