The National Hemophilia Foundation Is Highlighting Bleeding Disorders Awareness Month with New Campaign
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The National Hemophilia Foundation Is Highlighting Bleeding Disorders Awareness Month with New Campaign

In a recent press release, the National Hemophilia Foundation announced it has started a new campaign called “Start the Conversation” which will initiate conversations around inheritable blood disorders. Bleeding Disorders…

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Trialbee and ERGOMED Have Partnered to Recruit Patients for New Rare Disease Innovation Center
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Trialbee and ERGOMED Have Partnered to Recruit Patients for New Rare Disease Innovation Center

According to a recent article, Trialbee and ERGOMED have partnered to become the platform for patient recruitment for their new Rare Disease Innovation Center. Trialbee Trialbee is the leading platform…

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Adding Durvalumab to Chemotherapy Extends Survival in Patients with Advanced Biliary Tract Cancer
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Adding Durvalumab to Chemotherapy Extends Survival in Patients with Advanced Biliary Tract Cancer

According to a recent article, researchers have found that adding durvalumab to chemotherapy treatments results in prolonged survival rates in patients with advanced biliary tract cancer. Biliary Tract Cancer Biliary…

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EMA Recommends Changing Marketing Authorization for Avapritinib for Systemic Mastocytosis
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EMA Recommends Changing Marketing Authorization for Avapritinib for Systemic Mastocytosis

According to a recent article, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) is now recommending changing the marketing authorization for avapritinib, which is used…

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Study Shows Some People with Wet Age-Related Macular Degeneration May Be Able to Stop Eye Injection Therapy
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Study Shows Some People with Wet Age-Related Macular Degeneration May Be Able to Stop Eye Injection Therapy

  According to a recent article, a pilot study shows that around 30% of patients with wet age-related macular degeneration might have the ability to stop their eye injection therapy…

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New Research on Gene Therapies for Limb-Girdle Muscular Dystrophy 2B
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New Research on Gene Therapies for Limb-Girdle Muscular Dystrophy 2B

According to a recent article, Dr. Jyoti K. Jaiswal shared his research pertaining to gene therapies to be used to treat limb-girdle muscular dystrophy 2B (LGMD2B). Limb-Girdle Muscular Dystrophy (LGMD)…

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A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy
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A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy

  Betty Vertin recently wrote an article discussing her experience as a parent of a child with Duchenne muscular dystrophy. She shares the lessons she has learned in order to…

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Study Finds Blocking Sphingolipids Counteracts Muscular Dystrophy in Mice Models
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Study Finds Blocking Sphingolipids Counteracts Muscular Dystrophy in Mice Models

According to a recent article, a research team discovered that the inhibition of sphingolipid synthesis on mice models of Duchenne muscular dystrophy can counteract the certain symptoms of the disease.…

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MHRA Approves Two Phase 2 Clinical Trials of ‘1805 in Autoimmune Diseases in UK

According to a recent article, Revolo Biotherapeutics announced it has obtained approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for two Phase 2 clinical trials to analyze its…

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