World’s First Attempt at Human Head Transplantation for Rare SMA
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World’s First Attempt at Human Head Transplantation for Rare SMA

The line between genius and madness is often closer than people realize. Don’t tell Dr. Sergio Canavero that he can’t help his patient with spinal muscular atrophy (SMA). How many…

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Communication Could Mean Progress for Rare Diseases like Waldenstrom’s Macroglobulinemia
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Communication Could Mean Progress for Rare Diseases like Waldenstrom’s Macroglobulinemia

Communication in the medical field doesn’t happen as much as it should. But, when it does, great discoveries often occur. The thing is, science is complicated. Researchers have so much…

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This Big Sister Helped Save Her Little Brother Battling ROHHAD
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This Big Sister Helped Save Her Little Brother Battling ROHHAD

At 5 years old, Holton Jones was diagnosed with ROHHAD, or rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation. According to the Pensacola News Journal, his parents knew something…

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Enrollment Completed in Niemann-Pick Disease Study!
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Enrollment Completed in Niemann-Pick Disease Study!

The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type C disease. Niemann-Pick disease is a…

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My Challenging, Yet Rewarding Life, With Charcot-Marie-Tooth Disease

In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy held an essay contest for tickets to a Charcot-Marie-Tooth disease event. We asked members of the Charcot-Marie Tooth (CMT) community to tell…

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This Just In: More Support For Exciting New IPF Treatment!
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This Just In: More Support For Exciting New IPF Treatment!

Support for Ofev® just keeps coming! Ofev® (nintedanib) was first approved by the U.S. FDA in 2014 for the treatment of idiopathic pulmonary fibrosis (IPF) after clinical trials, especially the INPULSIS…

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For Multiple Myeloma Patients: Two Therapies are Better than One
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For Multiple Myeloma Patients: Two Therapies are Better than One

Results from the Phase 3 CASTOR trial from Janssen Pharmaceutical Companies were recently released, finding that Darzalex (daratumumab) was extremely effective at reducing the risk of multiple myeloma progression or…

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Umbilical Cord Blood Could Make a Difference in Sickle Cell Anemia Treatment
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Umbilical Cord Blood Could Make a Difference in Sickle Cell Anemia Treatment

A new public cord blood donation center recently opened up in New York. This is the second of this type of facility to open in the state. In the past,…

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This Organization Helps Those with SMA AND Holds Cool Concerts
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This Organization Helps Those with SMA AND Holds Cool Concerts

Adyn’s Dream is a charity in Athens, Ohio. The organization provides live events that benefit families and their children that have received a diagnosis of spinal muscular atrophy (SMA). The…

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