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

 

Dick DeVos; Giving Donations with No Strings Attached

Dick and his wife Betsy DeVos are a controversial couple in the world of philanthropy. This controversy originates from their enormous contributions to politics in the United States. During my research, I learnt that Dick and his wife Betsy had given donations amounting to $193 million. Nevertheless, as much as they are in the limelight for massive political donations, I discovered that much of their money goes to charitable causes as opposed to political ones.

 

For instance, Betsy revealed in her vetting process to become the US Education Secretary that the DeVos Family Foundation bequeathed $11.6 million to charitable ventures in 2015. This amount was twice more that they had donated to political campaigns for the past five years, $5.3 million. Therefore, I believe that they are genuine philanthropists and not led by political agendas.

 

Dick and Betsy are from families that have been actively involved in the Republican Party politics for many years. This also includes the couple’s four children who are also prominent philanthropists. In 2015, the DeVos family was ranked 24th on Forbes America’s Top Givers because of their total contributions amounting to $104 million in the same year. Moreover, I realised Forbes analysed their wealth only to reveal that the couple’s lifetime contributions approximated to 1.33 billion, which is a quarter if their entire fortune, $5.2 billion.

 

Dick and Betsy decided to reveal to the world the different causes they supported and the amount given to each cause in 2015. Education was a top priority for the high profiled couple who gave away $ 3 million to educational ventures and an extra $ 375,000 to societies that supported educational reforms. Other key areas included Public Policy ($1.3 million), Leadership and Development ($1.5 million, Health and Human services ($618,000), and Churches ($488,250).

 

About Dick DeVos

 

Dick DeVos attended the Northwood University for his undergraduate. He has had a vast career exposure working in countless executive posts at Amway, and NBA’s Orlando Magic. Currently, DeVos is the president and CEO of the Windquest Group. Beside business roles, Mr DeVos leads a number of community initiatives. For instance, I discovered that he is the founder of the Education Freedom Fund that has given put approximately 4,000 scholarships to disadvantaged children at the West Michigan Aviation Academy based in Michigan. DeVos has also sat on the State Board of Education.

 

Mr DeVos has won the National Champion sailor twice and is a qualified jet aircraft and helicopter pilot. Checking his Facebook page, I learnt that he is a public figure with over 18, 549 likes and has 18, 815 people following him. Most of his posts on this page include links to charitable ventures he is involved in, which are accompanied by several likes and comments. Mr DeVos has been married to Betsy for about 35 years, and they have four children and five grandchildren.