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

 

How Eva Moskowitz Became A Leader In The School Reform Movement

Eva Moskowitz has spent her professional career in the educational industry. She is the founder of Success Academy Charter Schools, a New York City chain of charter schools over which she leads as the Chief Executive Officer. She established the first Success Academy school in 2006 in the Harlem neighborhood. Eva Moskowitz’s leadership has led to there now being 32 schools that additionally located in Brooklyn, Manhattan, Queens, and the Bronx.

Serving 9,000 children that mostly come from low-income households, The Success Academy still outperforms even nearby affluent districts. Eva Moskowitz built a foundation at these schools that hold everyone accountable for achieving high educational standards, including the students themselves. The teacher’s work in a collaborative environment where they learn from each other the best practices in teaching children.

Eva Moskowitz was once the chair of the New York City Council Education Committee. She became a leader in school reform while investigating public schools and seeing how poorly they were run and their inability to properly educate children. During her time on the council, she exposed many scandals and problems at schools ranging from teacher practice to inadequate lighting in classrooms. She used the information she gathered while serving in this position to inform her choices at Success Academy in providing a better educational environment to properly foment learning.

Before founding the Success Academy, Eva Moskowitz was a professor at a number of universities including Columbia Unversity, Vanderbilt University, and the City University of New York. She earned her own college degrees at the University of Pennsylvania, where she received her bachelor of arts, and at John Hopkins University where she earned a Ph.D. in American History.

The ongoing crisis in education across the United States has become a well-known issue. Eva Moskowitz maintains that the problems aren’t what people usually point at, including poverty, African-Americans, and inner-cities. Rather, the problem is that the standards at public schools encourage mediocrity in both how teacher’s approach their material and the standards that students are held to. Her success at the Success Academy shows that every child can learn when given the opportunity.

To take a look at this businesswoman who is changing education like never before, take a look at her Wiki or Business.com pages.