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  1. www.fda.gov › drugs › our-perspectiveFDA Opens Doors for More Treatments for Rare Diseases

    5 days ago · FDA is taking steps to help accelerate the development of novel drugs and biologics through the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. Learn more.

  2. www.medscape.org › viewarticle › 968289_sidebar1Rare Disease 101: The Basics - Medscape Education

    3 days ago · The point prevalence of a rare disease is 3.5% to 5.9%, which equates to ~350 million people worldwide, which is the equivalent or almost of the population of the USA. The aggregated cost burden of rare disease is estimated to be greater than that of heart disease, stroke, and disability combined.

  3. www.medscape.org › sites › advancesPathways in Rare Disease - Medscape Education

    3 days ago · Pathways in Rare Disease: Being Rare Together Rare diseases affect approximately 300,000 patients worldwide. Yet, the journey to the right diagnosis may take an average 6 to 8 years with visits to multiple specialists and several misdiagnoses.

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  4. www.nature.com › articles › d41573/024/00114-3When chronic and rare disease worlds converge - Nature

    5 days ago · Sharon Barr, head of BioPharmaceuticals R&D at AstraZeneca, discusses her chronic disease research priorities, including the need for new diagnostic tools, fixed-dose drugs and a better ...

  5. scitechdaily.com › mystery-solved-surprisingMystery Solved: Surprising Origins for a Rare Cancer Uncovered

    5 days ago · A rare and currently incurable disease that attacks the liver of children, adolescents, and young adults, FLC is caused when a small deletion in chromosome 19 causes the fusion of two genes, a discovery made in 2014 in the lab of Rockefeller’s Sanford M. Simon, whose own then-teenage daughter, Elana, had not only been diagnosed with liver ...

  6. www.agrisafe.org › more-than-two-decades-of-nihMore than two decades of NIH research gives answers on a rare ...

    5 days ago · Today, clinicians have more effective treatments for this rare disease. And patients have more tools to get the help they need. What is fibrous dysplasia/McCune-Albright Syndrome? Fibrous dysplasia (FD) and McCune-Albright Syndrome (MAS) are separate conditions with the same genetic variation.

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  8. magazine.medlineplus.gov › article › more-than-twoMore than two decades of NIH research gives answers on a rare ...

    5 days ago · Decades of NIH research has improved clinicians’ understanding of and treatment for fibrous dysplasia/McCune-Albright Syndrome (FD/MAS), a rare disease that affects bones, skin, and the endocrine system. In particular, it can lead to weak or misshapen bones and can reduce patients’ quality of life. Researchers from the National Institute of Dental and Craniofacial Research and the National ...

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