Funds Raised For Researching Mucopolysaccharidosis and Other Rare Diseases

Xconomy.com has just released that funds have been raised for a variety of companies investigating therapies for rare conditions. Unfortunately, novel therapies and inventions cost million of dollars to develop. Thankfully,…

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A New Phase II Trial for Myelodysplastic Syndromes and Acute Myeloid Leukemia is Underway!
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A New Phase II Trial for Myelodysplastic Syndromes and Acute Myeloid Leukemia is Underway!

Myelodysplastic syndromes (MDS) is the name used to describe a group of stem cell disorders in which the body can't produce an adequate amount of platelets and blood cells. Currently,…

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Australian Government Initiates New Trial Program to Support Individuals Recently Diagnosed with a Rare Disease

Being diagnosed with any serious condition can be scary, overwhelming, and full of unknowns. For those who are diagnosed with a rare disease such as Duchenne muscular dystrophy, spinal muscular atrophy,…

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A Recap on 2018: Record Number of Drugs Approved by the FDA for Rare Diseases

Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…

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Positive Results for aTTP Announced by New England Journal of Medicine

Patients with acquired thrombotic thrombocytopenic purpura (aTTP) showed a significantly improved platelet count response after treatment with Cablivi® according to the New England Journal of Medicine's Phase 3 trial results. A…

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New Technology for Diagnosing Rare Conditions by Photograph May Foster Employment Discrimination?

As discussed in a recent article on PatientWorthy the FDNA has recently developed a new kind of algorithm which can diagnose rare genetic conditions such as Angelman syndrome, Williams syndrome, and Fragile X syndrome using…

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National Geographic Features Innovative Research That Will Advance Precision Medicine

Perhaps the most difficult thing about rare disease research is that not only do the conditions affect only a small portion of the population, but like every diagnosis, they affect…

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