More Evidence Suggesting Nusinersen Effective for Spinal Muscular Atrophy Types II and III
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More Evidence Suggesting Nusinersen Effective for Spinal Muscular Atrophy Types II and III

According to a press release from the American Academy of Neurology published by EurekAlert, new research published in Neurology further suggests that established spinal muscular atrophy (SMA) drug nusinersen could…

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Phase 2a Clinical Trial for Investigational Cell Therapy Now Recruiting Primary Sclerosing Cholangitis and Autoimmune Hepatitis Patients

Primary Sclerosing Cholangitis (PSC) and Autoimmune Hepatitis (AIH) are both rare and chronic liver diseases. They cause the bile ducts in the liver to become inflamed, resulting in severe liver damage.…

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Orphan Drug Designation Given to New Potential Oral Therapy for Hypereosinophilic Syndrome

Hypereosinophilic Syndrome Hypereosinophilic syndrome (HES) is a rare disease which causes high levels of white blood cells in the body. These cells are called eosinophils, and they're essential for proper…

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Study Indicates More Research is Needed on Transgender Acute Myeloid Leukemia Patients to Improve Patient Care

In a world that is just beginning to make space for the open discussion of transgender individual's life experiences, there's still a huge gap in the conversation regarding healthcare. This…

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Phenylketonuria Patient Didn’t Receive Life-Altering Treatment for 10 Years, Alludes to Bigger Problem
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Phenylketonuria Patient Didn’t Receive Life-Altering Treatment for 10 Years, Alludes to Bigger Problem

Within the pharmaceutical industry, we need 1) better policies for patients and 2) improved communication/education of the policies that do exist for patients and the rights that they have. Ultimately,…

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Experimental Treatment for Neuromyelitis Optica Earns Breakthrough Therapy Designation

According to a story from BioSpace, the biotechnology company Viela Bio recently announced that the US Food and Drug Administration (FDA) has awarded an experimental therapy in development by the…

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Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa

The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…

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Study Shows Hypertension Drug Could be Effective Treatment for Huntington’s Disease and Similar Illnesses

Neurodegenerative Diseases There are a wide range of neurodegenerative diseases including Huntington's disease and Parkinson's disease. These conditions typically affect the older population. As the world's population ages, more people…

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New Sensor Technology Can Detect Medication Response Automatically for Parkinson’s Disease Patients
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New Sensor Technology Can Detect Medication Response Automatically for Parkinson’s Disease Patients

According to a story from EurekAlert!, effective management of Parkinson's disease can be a serious challenge. Part of the reason that the management of the illness is so difficult is…

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Phase 3 Trial for Graft Versus Host Disease to Finally Begin After Funding Boost
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Phase 3 Trial for Graft Versus Host Disease to Finally Begin After Funding Boost

Background Novartis ended all of their gene therapy programs which were oncology related in 2016. Unfortunately, that meant an end to many programs that still had potential. This included cell…

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FDA Grants Orphan Drug Designation to Experimental Acute Lymphoblastic Leukemia Drug

According to a press release from British biopharmaceutical manufacturer Autolus Therapeutics, the American Food and Drug Administration (FDA) has granted the Company's experimental acute lymphoblastic leukemia (ALL) cell-based immunotherapy treatment…

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