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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.

GSK’s anti-BCMA drug leads crowded CHMP recommendations

GSK’s anti-BCMA drug leads crowded CHMP recommendations

Meanwhile, Sobi was handed a negative opinion for its drug Gamifant (emapalumab) as a treatment for the genetic disease primary haemophagocytic lymphohistiocytosis (HLH), which is characterised by an overactive immune system.

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Latest Intelligence

  • 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

  • A roadmap to the successful treatment of Alzheimer’s disease A roadmap to the successful treatment of Alzheimer’s disease

    Antioquia is home to the world’s largest concentration of people who carry a rare genetic mutation that makes them 100% certain to develop Alzheimer’s disease. ... In other words, there are genetic, lifestyle and environmental factors – to name a

  • 2019: CRISPR and therapeutic gene editing comes of age 2019: CRISPR and therapeutic gene editing comes of age

    genetic disease target. ... The phase 1/2 CHAMPIONS study did show that a single injection of SB-913 for the rare genetic disease mucopolysaccharidosis type II (MPS II) or Hunter syndrome was able 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. .

  • 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

  • OPEN Health’s virtual reflections on the World Orphan Drug Congress US OPEN Health’s virtual reflections on the World Orphan Drug Congress US

    these vital interventions that hold the promise of changing the face of the disease. ... We would be privileged to take a few steps towards making a positive change in the journeys of so many impacted by rare disease and genetic disorders.

  • Empowered patients: shaking the foundations of healthcare

    outcomes. We will be able to harness the data of every patient to better understand disease, ensuring our genetic databases are continuously updated. ... However, this is putting pressure on doctors to better understand the genetic foundations of disease.

  • Medical information in precision medicine

    The process of sequencing the DNA of tumours and zoning in on the genetic drivers of disease is magical and the people working behind the scenes are driving that magic every ... Treating patients down to the molecular level of their disease is incredibly

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