Review of Amicus Therapeutics Biopharmaceutical Achievement

Founded in 2002, Amicus Therapeutics is a public biopharmaceutical company that handles devastating rare and orphan illnesses. The firm focuses on disorders collectively referred to as Lysosomal Storage Disorders (LSDs). They include Pompe disease, Fabry disease, CDKL5, and rare genetic connective skin disorders called Epidermolysis Bullosa (EB).

 

Based in Cranbury, New Jersey, Amicus Therapeutics goal involves making the lives of the patients meaningful. The company collaborates with patients, health practitioners, and the caregivers to assist the rare disease community. They have utilized the aspect of science to invent and develop effective Enzyme Replacement Therapies (ERT). The company’s product is founded in a big way on the Chaperone-Advanced Replacement Therapy (CHART).

 

Amicus Therapeutics Chairman and CEO, John F. Crowley has been involved with biotechnology stems since 1998 after losing his two children with Pompe disease. John Crowley has dedicated his life to developing remedies for Pompe disease as well as other rare disorders. He has enabled the company to develop a technology called pharmacological chaperone that treat specific LSDs. This LSDs have traits of unfolded, unstable or misfolded mutated proteins. The technology is designed with tiny molecules which selectively target enzyme with the aim of stabilizing and turning it into the required three-dimensional shape.

 

The company’s leading product, though in the last stage of development, is referred to as Migalastat (https://finance.yahoo.com/quote/FOLD?ltr=1). The personalized medicine is meant to treat patients with Fabry disorder on the ground of individual genetic diagnosis. For the Epidermolysis Bullosa, Amicus Therapeutics is developing a first to market therapy called SD-101. Its topping biological program is the ATB200/AT2221, a different Pompe disease ERT that will be combined with the pharmacological chaperone.

 

Amicus Therapeutics in its development has collaborated with other companies as well acquiring others. In 2010, it worked with JCR pharmaceutical and GlaxoSmithKline to research on alpha-galactosidase. In 2013, it acquired a competitor, Callidus Biopharma as well as Scioderm in 2015.

Dr. Mark Holterman: Helping Support Dreams

Dr. Mark Holterman is many things. He is a professor of pediatrics at the University of Illinois College of Medicine, as well as CEO of Mariam Global Health. He has maintained both positions for the past five years.

 

As a medical specialist, Dr. Mark Holterman is passionate about a few organizations at support causes in the medical field. One organization that Dr. Holterman supports is the International Pediatric Specialists Alliance for the Children of Vietnam (IPSAC-VN).

 

The International Pediatrics Specialists Alliance for the Children of Vietnam’s goal is to improve pediatric medical treatment for the children of Vietnam. The organization works to provide necessary supplies and other supplies to medical personnel.

 

The IPSAC-VN also supports their local resident’s dreams of working in the medical field by providing scholarships through its International Scholar Program (Doctors.HealthGrove). The program allows them to further their education at select institutions in the United States.

 

The scholars are provided with funding for traveling to the United States, and they get hosted to gain experience in medical research or in clinical examinations at a medical institution. After the program ends, the scholars speak to supporters of the International Scholar Program about using the knowledge they gained to help their local medical community in Vietnam.

 

Dr. Mark Holterman is a pediatric surgeon at the Advocate Christ Children’s Hospital, and The Children’s Hospital Illinois, St. Francis Medical Center. Dr. Holterman is also a member of the American College of Surgeons and the American Academy of Pediatrics, amongst other organizations. He specializes in pediatric surgery and conducts research on regenerative medicines, stem cell therapies, obesity, and novel cancer treatments.

 

Dr. Mark Holterman earned his degree in biology from Yale University and his MD and Ph.D. from the University of Virginia (http://www.vitals.com/doctors/Dr_Mark_Holterman.html). He then went on to complete his residency at the University of Virginia Health Sciences focusing on general surgery. Also, completed a fellowship at the Children’s Hospital and Medical Center through the University of Washington in pediatric surgery.