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
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Our current portfolio spans four therapeutic areas, including 1) rare hematology, 2) rare endocrine/metabolic, 3) rare neurology and 4) rare cardiology. Now, we ...
Cure Rare Disease™ is developing advanced therapeutics for those who have been diagnosed with rare, genetic diseases that have no treatment or cures.
NORD works with partners in the patient community, government, academia, and industry who share the ultimate goal of identifying, treating, and curing rare ...
Feb 28, 2024 · An orphan drug is a drug intended for use in a rare disease. The World Health Organization defines a rare disease—sometimes referred to as ...
For 30 years, Alexion's pioneering science has led to the development of transformational medicines for rare diseases, and we continue to lead the way today.
Each Center offers world-class doctors in all major specialties and brings together medical teams experienced in diagnosing and treating a wide array of rare ...
Jul 13, 2021 · Treatments are often unavailable for rare disease patients, especially in low-and-middle-income countries. Reasons for this include lack of ...
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 ...