Zolgensma—which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay—is currently the most expensive drug in the world. A one-time treatment of the life-saving drug ...
Sep 1, 2021
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16
A new mechanism that stops the progression of Huntington's disease in cells has been identified by scientists at UCL and the University of Cambridge, as part of their research groups at the UK Dementia Research Institute.
Aug 31, 2021
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118
While most cases of type 1 diabetes are classified as autoimmune diseases, a minor proportion of these patients have non-immune-mediated type 1 diabetes, sometimes referred to as type 1 B diabetes. Researchers from Children's ...
Aug 27, 2021
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255
The monoclonal antibody lanadelumab has been approved in Europe since 2015 as a long-term prophylaxis to prevent attacks of hereditary angioedema. Since this is a rare disease, the Federal Joint Committee (G-BA) initially ...
Aug 20, 2021
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2
Scientists at Oregon Health & Science University have identified a naturally occurring mutation in nonhuman primates that closely resembles a rare neurodegenerative disease in people.
Aug 6, 2021
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394
France's pharmaceutical giant Sanofi, which has lagged behind rivals in developing new generation mRNA Covid-19 vaccines, on Tuesday said it has purchased a US firm specialising in the technology.
Aug 3, 2021
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142
British pharmaceuticals giant AstraZeneca said Thursday that its COVID jab generated $1.2 billion (1.0 billion euros) in sales in the first half of the year as global vaccination programmes accelerated.
Jul 29, 2021
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123
Roughly 1 out of 15,000 people born suffer from Facioscapulohumeral Muscular Dystrophy (FSHD), a rare disease that starts with the progressive weakening of facial muscles and then the shoulders and upper body. By the time ...
Jul 20, 2021
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5
A phase II study led by researchers from The University of Texas MD Anderson Cancer Center found that treatment with atezolizumab and bevacizumab was well-tolerated and resulted in a 40% objective response rate in patients ...
Jul 14, 2021
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5
A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led by researchers at The Ohio State University ...
Jul 12, 2021
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