- Home
- Equipment
- usa massachusetts
- rare disease day 2017 with a commitment to provide new insights into rare diseases
Show results for
Refine by
Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Equipment Supplied In Usa Massachusetts
3 equipment items found
by:Cartesian Therapeutics based inGaithersburg, MASSACHUSETTS (USA)
Selecta’s first ImmTORTM + gene therapy candidate MMA-101 for methylmalonic acidemia (MMA) is expected to enter clinical trials the end of 2021. MMA is a rare metabolic disease that may lead to metabolic acidosis and hyperammonemia and is associated with long-term complications including feeding problems, developmental delay, intellectual disability, and chronic kidney ...
by:Alexion Pharmaceuticals, Inc. based inBoston, MASSACHUSETTS (USA)
People with rare diseases often wait years to receive a proper diagnosis and many never receive one at all. RARE ANSWERS™ is a system of innovative and sustainable tools designed to help shorten the diagnostic journey and time to initiating treatment of children with a rare disease. Developed in collaboration with leading children’s hospitals and technology and data-science ...
by:Aegle Therapeutics based inWoburn, MASSACHUSETTS (USA)
Epidermolysis bullosa (“EB”) is a group of rare genetic disorders that manifests as blistering or erosion of the skin in response to little or no apparent trauma. There are many genetic and symptomatic variations of EB. EB is always painful, often pervasive and debilitating, and is in some cases lethal before the age of 30. EB affects both genders and every racial and ethnic ...