Clinical Trial Updates for Achondroplasia and Hemophilia A!
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Clinical Trial Updates for Achondroplasia and Hemophilia A!

BioMarin has just released an update on their achondroplasia and hemophilia clinical trials. Exciting things are in the works! Achondroplasia Update Achondroplasia causes short stature. A phase 3 trial working to…

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Bioverativ Presents Updates on Treatments and Research for Sickle Cell Anemia and Hemophilia
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Bioverativ Presents Updates on Treatments and Research for Sickle Cell Anemia and Hemophilia

According to a story from Business Wire, the drug developer Bioverativ, Inc., recently announced its intention to present data related to its latest developments in blood disorder therapies. This data…

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Rare Disease and Personal Identity: Why This Hemophilia Patient Isn’t Interested in a Cure
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Rare Disease and Personal Identity: Why This Hemophilia Patient Isn’t Interested in a Cure

According to a story from The Atlantic, the continual development of gene editing technologies like CRISPR have the potential to cure a substantial portion of rare diseases that are linked…

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Cryo-Electron Imaging Provides New Info About Virus Vectors in Gene Therapy
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Cryo-Electron Imaging Provides New Info About Virus Vectors in Gene Therapy

According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…

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Cell Encapsulation Technique That Could Treat Glaucoma and Hemophilia May Make Insulin Injections a Thing of the Past

  According to Science Blog, a therapeutics company is on its way to creating an implantable, insulin-producing device that may give injections a run for their money. Therapies similar to…

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Is Fetal Gene Therapy the Cure to These Rare Diseases?
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Is Fetal Gene Therapy the Cure to These Rare Diseases?

Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease.  With fetal gene…

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Is There a Genetic Condition in Your Family?
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Is There a Genetic Condition in Your Family?

Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…

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FDA Commissioner Scott Gottlieb Issues Statement With Gene Therapy Guidelines

According to a story from the U.S. Food and Drug Administration (FDA), Commissioner Scott Gottlieb has released a press release which discusses in detail the agency's efforts in the development…

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ICYMI: National Hemophilia Foundation Announces Collaboration on Von Willebrand Disease Guidelines
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ICYMI: National Hemophilia Foundation Announces Collaboration on Von Willebrand Disease Guidelines

According to a June 20th report by the National Hemophila Foundation (NHF), the NHF along with several other prominent organizations in the bleeding disease community will be joining forces to…

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First Hemophilia B Patient Enrolled in Potential Breakthrough Phase III Trial

According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…

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This New Program Can Help Make Life Easier for Rare Disease Patients
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This New Program Can Help Make Life Easier for Rare Disease Patients

According to a story from CheckOrphan, Express Scripts has prepared a Rare Conditions Care Value (RCCV) program. This program is designed to help make the diagnosis and treatment process for…

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Hemophilia Doesn’t Just Affect Men, Let’s Stop Talking like It Does
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Hemophilia Doesn’t Just Affect Men, Let’s Stop Talking like It Does

Hemophilia can affect anyone. Let’s make a few things clear: Women are carriers of hemophilia and pass the gene, but men determine the child’s gender. It’s not all on you,…

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Tips for a New Hemophilia Patient and New Caregiver: Reporting from the WFH Congress
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Tips for a New Hemophilia Patient and New Caregiver: Reporting from the WFH Congress

To the patient: Accepting your diagnosis doesn’t mean you can’t be mad about it. It simply means you understand that the reality of your life has changed and you know…

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A Letter to Parents on Transitioning from Pediatric to Adult Care in Hemophilia: Reporting from the WFH Congress
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A Letter to Parents on Transitioning from Pediatric to Adult Care in Hemophilia: Reporting from the WFH Congress

Dear Parents, You need to understand that you are taking on a completely new role in your child’s life. You will no longer be controlling their hemophilia for them. It’s…

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Express Scripts Makes Diagnosis Easier with Second Opinion
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Express Scripts Makes Diagnosis Easier with Second Opinion

According to PRNewswire, Express Scripts announced a new program to benefit people living with rare diseases on May 31st. The program, called Second Opinion is part of the company’s larger…

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