Small molecule medications, antibody therapy, enzyme replacement therapy, gene therapy, stem cell therapy, and drug repositioning are some of the modalities used to treat rare diseases.
Oct 12, 2022
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Dec 13, 2022 · Many rare conditions are life-threatening and most do not have treatments. Drug, biologic, and device development in rare diseases is ...
Rare Disease: Pioneering the Science of Breakthroughs - Pfizer
www.pfizer.com › science › focus-areas
Our current portfolio spans four therapeutic areas, including 1) rare hematology, 2) rare endocrine/metabolic, 3) rare neurology and 4) rare cardiology. Now, we ...
NORD works with partners in the patient community, government, academia, and industry who share the ultimate goal of identifying, treating, and curing rare ...
May 12, 2022 · The FDA works to enhance to the availability of treatments for rare diseases by evaluating information from product sponsors to determine if ...