This Failed Clinical Trial Helped Researchers Learn More About Sickle Cell Disease
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This Failed Clinical Trial Helped Researchers Learn More About Sickle Cell Disease

  No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…

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Planning for Phase 3 for Study of Treatment for NASH Has Begun
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Planning for Phase 3 for Study of Treatment for NASH Has Begun

  Galectin Therapeutics has recently started to plan the third phase of its clinical trial of belapectin, which treats nonalcoholic steatohepatitis (NASH). They are making refinements based on a conversation…

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The Relationship Between Asthma and Alpha-1 Antitrypsin Deficiency
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The Relationship Between Asthma and Alpha-1 Antitrypsin Deficiency

  Alpha-1 antitrypsin deficiency (A1AD) shares similarities with asthma, which leads to it often being misdiagnosed as asthma. This misdiagnosis leads to insufficient treatment. Toronto Western Hospital's Asthma and Airways…

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Ganaxolone Being Investigated as Treatment for Tuberous Sclerosis and CDKL5 Deficiency Disorder
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Ganaxolone Being Investigated as Treatment for Tuberous Sclerosis and CDKL5 Deficiency Disorder

Marinus Pharmaceuticals is a pharmaceutical company that works to create treatments for epilepsy and other neuropsychiatric disorders. They are in the process of conducting clinical trials to evaluate the safety…

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XPOVIO Being Evaluated for Treatment of Diffuse Large B-Cell Lymphoma
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XPOVIO Being Evaluated for Treatment of Diffuse Large B-Cell Lymphoma

Karyopharm, a pharmaceutical company based in Newton, Massachusetts, has submitted a New Drug Application to the FDA for their drug XPOVIO, which is meant for the treatment of diffuse large…

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Study: Odiparcil Shows Positive Results in Treatment of Mucopolysaccharidosis Type VI (MPS VI)
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Study: Odiparcil Shows Positive Results in Treatment of Mucopolysaccharidosis Type VI (MPS VI)

Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the…

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Study: Belantamab Mafodotin Shows Positive Results in People with Multiple Myeloma
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Study: Belantamab Mafodotin Shows Positive Results in People with Multiple Myeloma

Copyscape score: 4% Cancer is a disease that affects different people in different ways, and for some people, no existing treatments can effectively treat their cancer. GlaxoSmithKline has recently released…

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Alnylam Seeks FDA Approval for First Treatment of Primary Hyperoxaluria Type 1

Copyscape score: 2% Alnylam has recently completed the third phase of their clinical trial of lumasiran, which was created for the treatment of primary hyperoxaluria type 1 (PH1). While they…

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A Woman with Sickle Cell Disease Hopes to Become a Doctor to Help Others with the Same Disease

Copyscape score: 9% Deondra Clark was unable to have many of the experiences that other children had. Homecoming, prom, sports, and other childhood activities were not a possibility for her,…

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Catherine’s Story: Myalgic Encephalomyelitis Makes Her Constantly Tired
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Catherine’s Story: Myalgic Encephalomyelitis Makes Her Constantly Tired

For a long time, Catherine Allen went without a diagnose to explain the myriad of symptoms that she experienced. Doctors dismissed her experiences as common feelings in teenagers, when in…

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Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency
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Gene Therapy Being Assessed for Treatment of X-Linked Severe Combined Immunodeficiency

  Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…

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Experimental Myelodysplastic Syndrome Treatment Receives Breakthrough Therapy Designation

  A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an…

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27th UEG Week: Anti-TNF-α Therapy as a Treatment for Inflammatory Bowel Disease
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27th UEG Week: Anti-TNF-α Therapy as a Treatment for Inflammatory Bowel Disease

  The 27th United European Gastroenterology Week took place in Barcelona, Spain in October, at which some of the leading experts in gastroenterology spoke about their field. One of the…

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FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency
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FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency

Dicerna Pharmaceuticals works in ribonucleic interference (RNAi) therapies as treatments of rare diseases. They have recently been granted the orphan drug designation by the European Commission for their drug, DCR-A1AT.…

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