Notizia

Rare diseases are mostly serious, chronic and life-threatening, associated with both psychological as well as financial burdens. In addition, only a few of them have effective drug treatment available. The European Union (EU) definition of a rare disease is one that affects fewer than 5 in 10,000 people. These affe...

A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government g...

CRISPR treatment delivers benefits in mice The researchers from Duke University along with drugmaker Sarepta Therapeutics published a report stating that a single change in the genetic system of Duchenne muscular dystrophy (DMD) affected mice, using CRISPR gene editing tool resulted in increasing their life spa...

Neurogene raises USD 68.5 Million for rare neurological diseases Neurogene netted USD 68.5 million to advance multiple gene therapy programs in its series A round. The idea is to take its major programs into IND-enabling studies for Aspartylglucosaminuria (AGU) and a rare form of Charcot-Marie Tooth disease with...

https://www.slideshare.net/DelveInsight/myasthenia-gravis-126250701?qid=b9adbccf-6d91-4e5e-84e1-7603c87a029e&v=&b=&from_search=1 Myasthenia gravis (MG) is an autoimmune syndrome caused by the failure of neuromuscular transmission, which results from the binding of autoantibodies to proteins involved i...

https://www.slideshare.net/DrBKAgrawal/neuroendocrine-tumors-nets?qid=c34d63bb-8495-4c65-9f4e-9348d299ed70&v=&b=&from_search=1 Neuroendocrine tumors (NETs) consists of a complex group of tumors that arise from cells called neuroendocrine cells. For more, view the presentation. For sample repor...