The FDA Has Awarded Orphan Drug Designation to a Therapy for Complications After Haematopoietic Cell Transplantation
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The FDA Has Awarded Orphan Drug Designation to a Therapy for Complications After Haematopoietic Cell Transplantation

The United States Food and Drug Administration has awarded Orphan Drug Designation to an experimental PLX cell therapy (called PLX-R18) as a treatment for graft failure and incomplete haematopoietic recovery…

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Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia
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Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia

According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…

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Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation
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Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation

According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…

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The FDA has Awarded Over $18 Million for Rare Disease Research
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The FDA has Awarded Over $18 Million for Rare Disease Research

The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news,…

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Clinical Trial Data Looking Promising for an Experimental Amyotrophic Lateral Sclerosis Treatment
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Clinical Trial Data Looking Promising for an Experimental Amyotrophic Lateral Sclerosis Treatment

According to a story from BioSpace, the biotechnology company BioElectron recently announced that the results from its Phase 2a clinical trial of its experimental product EPI-589 are looking positive. EPI-589…

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Cambridge Researchers Find That Gene Editing Could Help Treat Mitochondrial Diseases

According to a story from UPI, gene editing technology could be a potential treatment for mitochondrial diseases, a rare, genetic illness that is usually fatal. In a proof-of-concept experiment, researchers…

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Experimental Treatment Shuts Down Hereditary Angioedema Attacks Quickly in Trials
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Experimental Treatment Shuts Down Hereditary Angioedema Attacks Quickly in Trials

According to a story from Angioedema News, an investigational product from BioCryst Pharmaceuticals called BCX7353 was able to quickly subdue swelling attacks in hereditary angioedema in clinical trials. In addition,…

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Experimental Treatment for Peripheral T-Cell Lymphoma Gets Orphan Drug Designation
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Experimental Treatment for Peripheral T-Cell Lymphoma Gets Orphan Drug Designation

According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration…

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Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared
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Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared

Sangamo Therapeutics has shared details of their preliminary data from a clinical trial of the investigational drug SB-525 in patients with Haemophilia A. For more detailed information, you can read…

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Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…

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Genetic Condition Observed Only in Mice Discovered in First Patient
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Genetic Condition Observed Only in Mice Discovered in First Patient

According to a report by medicalexpress.com, researchers recently discovered a new genetic condition. Though researchers previously described the condition in animals, no human evidence had yet been found. Michigan State…

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A Group of Disorders Share Similarities in Their DNA 3D Folding Patterns, Researchers Say

Researchers at the University of Pennsylvania have studied the genetics of a class of neurological disorders that includes Huntington’s disease and Fragile X Syndrome. They found that there were similarities…

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A Boy With Congenital Muscular Dystrophy is Being Supported in a Special Way

Michael Dickerson, aged 42, is originally from Hoedspruit Limpopo, South Africa. Recently, Michael was participating in a series of long distance trail runs when he learned about the story of Daniël…

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ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment
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ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment

According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…

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Experimental Treatment for Ulcerative Colitis Shows Promise in Trials
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Experimental Treatment for Ulcerative Colitis Shows Promise in Trials

According to a story from Business Wire, the biotechnology company Abivax recently released the results from its Phase 2a clinical trial. This trial was testing its investigational product ABX464 as…

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