This page shows the latest Genetic disease news and features for those working in and with pharma, biotech and healthcare.
Affecting approximately 100, 000 people in the US, SCD is a life-long, incurable genetic disease causing red blood cells to take a distinct crescent shape, which can block blood vessels ... The disease can cause serious health problems, including anaemia,
Pompe disease is a rare genetic disorder that causes progressive weakness to the heart and skeletal muscles. ... MZE001, designed and developed by Maze, is an oral GYS1 inhibitor that aims to address Pompe disease by limiting glycogen accumulation.
The genetic disease affects approximately 100, 000 people in the US alone. ... Affecting approximately 100, 000 people in the US, SCD is a life-long, incurable genetic disease causing red blood cells to take a distinct crescent shape, which can block
XLH is a life-long genetic disease that causes abnormalities in the bones, muscles and joints. ... The Scottish Medicines Consortium (SMC) has enabled access to the first treatment addressing the underlying cause of X-linked hypophosphataemia (XLH), a
The rare genetic disorder affects around one in every 40, 000 people in the UK. ... Affecting approximately one in every 40, 000 people in the UK, Pompe disease is a rare genetic disorder caused by low levels of the enzyme acid alpha-glucosidase,
creating milestones, including the planned submission and subsequent FDA review of our biologics licensing application for lovo-cel for sickle cell disease.”. ... Beta-thal is a rare genetic blood disease caused by a gene defect that impairs the
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systems have the potential to edit and correct faulty genes, preventing them from causing disease. ... The application of this technology is clear in monogenic diseases and its potential is now being investigated for idiopathic diseases without a known
By Will Maier. Rare disease drug development poses special challenges that can be overcome by using real-world evidence (RWE). ... Disease natural history describes the demographic, genetic, environmental and other variables (eg treatment modalities,
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
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
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
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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
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
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
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
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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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.
Our client wanted to better understand the needs, preferences, and treatment gaps among adult patients with a rare genetic disease. ... Our client wanted to better understand the needs, preferences, and treatment gaps among adult patients with a rare
Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. ... patients and families affected by devastating diseases
Research shows that 99% of people carry at least one genetic variation that can affect their response to certain drugs including commonly prescribed painkillers, heart disease treatments, and antidepressants. ... While the upfront cost of this genetic
Batten Disease is a group of fatal genetic disorders. There are 13 types. ... There is no cure for these disorders but a treatment for CLN2 disease has been approved.
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