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

 

Joshua Tree: A Case Study in Conservation and Appreciation

Joshua Tree National Park stands apart from the other protected wildernesses of the California State Park system, simply because of its location. It sits at the intersection of three distinct ecosystems – the Mojave Desert, the Colorado Desert and the Little San Bernadino Mountains – in a protected space larger than the state of Rhode Island. From the high tablelands of the Mojave to the lower, warmer cactus forests in the eastern preserves, no other park in the state can boast the same diversity of ecosystems. Its standalone nature is symbolized in the Joshua trees from which it gets its name: the Joshua tree is confined almost exclusively to the Mojave and Joshua Tree National Park. Learn more: https://au.pinterest.com/wild_ark/

While a popular spot for camping, hiking, and astronomy, in recent times Joshua Tree’s popularity has increased through rock climbing. The rock formations of the park consist of blocks of granite strewn across the valleys, some exceeding two hundred feet in height. With such colorful names as “Giant Marbles”, “Old Woman Rock” and “North Horror Rock”, the boulders provide thousands of climbing routes at all levels of experience; the formations are especially popular with the rock climbing community during the winter months when other climbing sites like Yosemite are snowbound. Learn more: http://wildark.com/news/

The overall effect of the Joshua Tree National Park, from its colossal formations of stone to its rugged, solitary Joshua trees, is that of an alien landscape untouched by the modern world. Nowhere else on the planet looks quite like it.

WildArk, formed with the mission to conserve, protect, and research the various wild places of the world, is dedicated to a similar appreciation of the natural world that the Joshua Tree National Park provides. Through travel recommendation, wildlife conservancies and eco-journalism, WildArk hopes to foster a similar admiration of the natural world that is so well exemplified by the conservation efforts of the California State Park system in Joshua Tree. Its balance of conservation, appreciation and human enjoyment provides a perfect example of the environmentally-conscious mindset and framework that WildArk maintains, in its work in Africa and around the globe. Learn more: http://www.huffingtonpost.com.au/2016/10/20/mick-fanning-the-conservationist-first-look-at-his-expedition-t/

 

cited works

https://www.nps.gov/jotr/index.htm