Luspatercept Demonstrates Further Benefits in Transfusion-Dependent Beta Thalassemia
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Luspatercept Demonstrates Further Benefits in Transfusion-Dependent Beta Thalassemia

According to a story from Hematology Advisor, the drug luspatercept (marketed as Reblozyl) recently demonstrated its ability to reduce transfusion burden in patients with beta thalassemia that depend on regular…

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A Potential Treatment for hATTR Amyloidosis has Earned Fast Track Designation
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A Potential Treatment for hATTR Amyloidosis has Earned Fast Track Designation

According to a story from Biospace, the RNAi therapeutics company Alnylam Pharmaceuticals Inc. recently announced that its experimental product candidate vutrisiran has earned Fast Track designation from the US Food…

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Gene Therapy Could Cure Irreversible Blindness for X-Linked Retinitis Pigmentosa
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Gene Therapy Could Cure Irreversible Blindness for X-Linked Retinitis Pigmentosa

As reported in Biospace, a new gene therapy has made notable progress towards curing x-linked retinitis pigmentosa, a disease responsible for sight loss in men. Until now, the disease irreversibly…

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FDA Approves a Treatment for Neurofibromatosis Type 1 for the First Time Ever
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FDA Approves a Treatment for Neurofibromatosis Type 1 for the First Time Ever

A press release from the US Food and Drug Administration (FDA) recently announced the approval of the drug selumetinib (marketed as Koselugo) for the treatment of patients aged two years…

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FDA Accepts NDA for a Potential Pulmonary Arterial Hypertension Treatment
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FDA Accepts NDA for a Potential Pulmonary Arterial Hypertension Treatment

According to a story from globenewswire.com, the biopharmaceutical company Liquidia Technologies, Inc. just announced that the company's New Drug Application (NDA) has been officially accepted by the US Food and…

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ALUNBRIG Approved by European Commission as Treatment for Patients with ALK+ NSCLC
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ALUNBRIG Approved by European Commission as Treatment for Patients with ALK+ NSCLC

  According to a release on Biospace, the European Commission (EC) has approved Takeda Pharmaceutical's ALUNBRIG (brigatinib) as treatment for patients with anaplastic lymphoma kinase-positive (ALK+) advanced non-small cell lung cancer…

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Parkinson’s Disease:  A New Study Focusing on Preventing the Loss of Dopamine Producing Neurons
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Parkinson’s Disease:  A New Study Focusing on Preventing the Loss of Dopamine Producing Neurons

  The drug levodopa and most other therapies developed over the years for Parkinson's disease have been centered on correcting the lack of dopamine. Researchers acknowledge that the reason dopamine…

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Emerald Health Pharmaceuticals Initiates Phase 2 Clinical Study on Systemic Sclerosis
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Emerald Health Pharmaceuticals Initiates Phase 2 Clinical Study on Systemic Sclerosis

  A news release from Benzinga shares that biotechnology company Emerald Health Pharmaceuticals is beginning a Phase 2 clinical study on EHP-101 as a treatment for patients with systemic sclerosis.…

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Company Starts Compassionate Use Program for an Experimental COVID-19 Treatment
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Company Starts Compassionate Use Program for an Experimental COVID-19 Treatment

According to a story from gurufocus.com, the biotechnology company Capricor Therapeutics has recently announced the initiation of a compassionate use program for its experimental drug CAP-1002. This drug will be…

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Study Confirms Effectiveness of Gamma Knife Surgery for Intracanalicular Vestibular Schwannoma
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Study Confirms Effectiveness of Gamma Knife Surgery for Intracanalicular Vestibular Schwannoma

According to a study published in January in the scientific journal Acta Neurologica Scandinavica, the use of gamma knife surgery as a treatment for a rare tumor called a vestibular schwannoma can…

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