Amicus Therapeutics For Genetic Diseases

Amicus Therapeutics is highly passionate about its work. It is a global company in the field of biotechnology. This company is into advanced therapies for treating rare as well as orphan diseases. The company has a highly robust development pipeline that includes treatments for several genetic diseases in humans.

Migalastat is considered by Amicus Therapeutics as its lead product candidate (MarketWatch). This can be considered as personalized medicine. It is in the last stages of its development. This will be used for treating individuals who are suffering from Fabry disease. This would require genetic diagnosis. Another product candidate is SD-101. Even this is in its last stages of development. This will be the first therapy to be marketed. It can be used for the genetic connective tissue disorder known as Epidermolysis Bullosa (EB). Amicus Therapeutics is making use of its biologics along with the Chaperone-Advanced Replacement Therapy platform technologies. These are being used for developing several enzyme replacement therapy products. These can be used for Fabry disease, besides Pompe disease, as well as several other Lysosomal Storage Disorders.

The lead biologics program of Amicus Therapeutics is ATB200/AT2221. This is a Pompe disease ERT. It is to be administered along with a pharmacological chaperone.

Amicus Therapeutics offers three clinical programs. In addition, they can leverage their biologics capabilities along with platform technologies that can help in expanding their pipeline.

The biologics capabilities of Amicus Therapeutics helps to provide a unique tool that can be used for developing Enzyme Replacement Therapy products that can be used for LSDs. Presently, Amicus Therapeutics has novel ERTs already that can be used for Fabry as well as Pompe disease. Now the technology platforms can be used for creating future opportunities that can lead to novel ERT being developed for other LSDs.

Currently, Amicus Therapeutics is researching a potential protein replacement therapy. This will be used in preclinical studies for the CDKL5 deficiency. Any kind of genetic mutations on this CDKL5 gene can lead to CDKL5 protein deficiency. This disorder manifests itself as persistent seizures. These begin in infancy itself. It can lead to a severe impairment that can take place in neurological development.
More here :  https://www.crunchbase.com/organization/amicus-therapeutics

 

Jason Hope Knows the Future of Business Technology

If a company wants to remain competitive in today’s market they must know how to effectively compete. Companies compete on many different levels. One the most important ways for a business to maintain its edge over the competition is by implementing a solid technology system. Without solid technology platform to provide the best way to deliver business services and products; a business will not be able to operate at its best.

Jason Hope is a business guru that understands how important technology is for any business. He is a technology guru that provides consultation services to organizations about the best technology trends for their operations. Hope strongly endorses the use of the internet of things. The internet of things is a concept that describes how various different machines, electronics and devices can be connected together through the use of the world wide web.

In business, this is a valuable technology platform that will make it easier for people do business within society. Consumers can use this technology to purchase products from their refrigerator when they run out of a certain food. They could even purchase more bathroom supplies by sending out an order from their computerized toilet. While this might seem unusual; it is not and more information click here.

The concept of the internet of things is already being implemented. Manufacturers are producing more appliances, machines and devices with the ability to connect through the internet. Businesses are now understanding how valuable this technology is. They can use it to provide market niche products to consumers who need them the most.

Businesses can also use the internet of things to allow consumers to make payments through their smart watches and even their automobile. This will allow businesses to provide people with more payment options. It will also help a company to sell more products and services. Jason Hope is really excited by this technology. He knows that it will improve the quality of life for everyone and Jason’s lacrosse camp.

More visit: http://jasonhope.com/blog/