Amicus Therapeutics is a public American biopharmaceutical firm in Cranbury, New Jersey. This international biotechnology company is at the forefront of advanced pharmaceutical therapies that treat a broad range of orphan and rare, devastating diseases.

Behind the walls of Amicus Therapeutics are many successful medical programs which are as follows;

 

Amicus Therapeutics Programs

Amicus is science driven, and that’s why it’s able to deliver unmatched benefits to patients. This pharmaceutical firm works on the principals that their platform technologies and their advanced product line uniquely positions them at the front of therapies that address unmet needs for rare diseases.

Below are programs currently plans being worked on at Amicus Therapeutics

 

FABRY DISEASE PROGRAM

This program deals with the Fabry disease which is as a result of mutations in the GLA gene. These mutations cause alpha-Gal A, to be deficient or absent in the system.

In the absence of alpha-Gal A, the substrate lyso-Gb 3 and GL-3 accumulate leading to deterioration of cells within the affected areas of the individual’s body thus causing the different pathologies seen in Fabry disease.

At Amicus Therapeutics is where Fabry disease was found to be the cause of progressive organ damage of the renal, nervous, cardiac systems, as well as multiple other tissues.

 

Epidermolysis Bullossa Program

EB is a genetic disorder that presents itself as an erosion of the skin. In some cases, Epidermolysis Bullossa can be fatal and patients who suffer from this disease experience painful wounds which are infectious.

 

POMPE DISEASE

Another disease conquered by Amicus Therapeutics in their numerous programs is the Pompe disease. This rare condition is inherited LSD (Lysosomal Storage Disorder) and caused by lack of enzyme acid alpha-glucosidase (GAA). The absence of GAA leads to accumulation of glycogen in the muscles and specific tissues (Yahoo Finance). Continuous accumulation of this substrate leads to respiratory insufficiency and muscle weakness.

 

Preclinical Programs

Under this program, Amicus Therapeutics is researching a potential protein replacement therapy for individuals who suffer from CDKL5. The cyclin-dependent kinase-like 5 (CDKL5) is a gene on the X-chromosome that regulates several proteins and ensures healthy brain development. Most children who suffer from a lack of CDKL5 cannot care for themselves as they experience visual impairment, scoliosis, and gastrointestinal complications and sensory issues.

More about Amicus Therapeutics at https://www.indeed.com/q-Amicus-Therapeutics-l-Cranbury,-NJ-jobs.html


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