BioMarin Holds Clinical Study to Determine Antibodies that Might Impact Gene Therapy
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BioMarin Holds Clinical Study to Determine Antibodies that Might Impact Gene Therapy

Last month, biotechnology company BioMarin sought approval for an investigational gene therapy (valoctocogene roxaparvovec) for patients with hemophilia A. However, their approval was denied. Instead, the company was told that…

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Recall of Bleeding Disorder Drug Leaves Patients with Unanswered Questions
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Recall of Bleeding Disorder Drug Leaves Patients with Unanswered Questions

According to a story from hemophilia.org, the drug company Ferring Pharmaceuticals issued a recall of the nasal spray drug desmopressin (marketed as Stimate) on July 21st, 2020. This product is…

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Researchers Produce End-to-End X Chromosome DNA sequence

  As society becomes increasingly technological, researchers are getting closer and closer to mapping the entire human genome. In facts, shares Medical XPress, we may be nearly there. While many…

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Mouse Model of Gene Therapy is Shown to be Effective Against Hemophilia A
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Mouse Model of Gene Therapy is Shown to be Effective Against Hemophilia A

By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…

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New Clinical Expectations Needed to Treat Hemophilia
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New Clinical Expectations Needed to Treat Hemophilia

The medical realm is changing - but how does that impact patient care and clinical expectations? The National Hemophilia Foundation discusses how clinical expectations for patients with hemophilia don't necessarily…

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Certain Proteins May Predict Joint Bleeding in Patients with Hemophilia
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Certain Proteins May Predict Joint Bleeding in Patients with Hemophilia

  Patients with hemophilia may experience sudden bleeding, joint pain and inflammation, and deep bruising. But according to Hemophilia News, two proteins may be the cause. Researchers found that patients…

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Review of Rare Movement Disorders in India and the United States

A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…

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Rare Disease Advocates in India are Tirelessly Pushing for New Policies
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Rare Disease Advocates in India are Tirelessly Pushing for New Policies

Rare disease advocates in India are pushing for the Health Ministry to create a new fund for rare diseases. Additionally, they're pushing for medicines and medical equipment used for these…

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The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others
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The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others

The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…

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AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials
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AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials

AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina.  This company is dedicated to the research of gene therapies as potential therapeutic options…

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With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action
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As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action

According to a story from newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…

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