Taking It One Step Further in the Quest to Prolong Survival in Acute Myeloid Leukemia
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Taking It One Step Further in the Quest to Prolong Survival in Acute Myeloid Leukemia

  Dr. Lindsay Wilde, an oncologist at the Hematologic Malignancies Division of Jefferson University, recently contributed to an article in the Oncology Times outlining her research team’s new study and…

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ICYMI: Recurrent Respiratory Papillomatosis Treatment Receives Orphan Drug Designation
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ICYMI: Recurrent Respiratory Papillomatosis Treatment Receives Orphan Drug Designation

INO-3107, a treatment for recurrent respiratory papillomatosis (RRP), has recently received the Orphan Drug Designation from the FDA. This treatment is currently being evaluate in a Phase 1/2 trial by…

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Studies from UCSF Offer New Perspective on Fibromyalgia and Chronic Fatigue Syndrome
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Studies from UCSF Offer New Perspective on Fibromyalgia and Chronic Fatigue Syndrome

  Researchers discovered surprising results after performing new studies of chronic fatigue syndrome (ME/CFS) and fibromyalgia (FM). According to a report by Cort Johnson in HealthRising, the new studies give…

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Rare Pediatric Disease Designation Given for Investigative Lymphatic Malformations Therapy
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Rare Pediatric Disease Designation Given for Investigative Lymphatic Malformations Therapy

Rare Pediatric Disease Designation has just been granted by the FDA for an investigative therapy for lymphatic malformations (LMs). This therapy is called TARA-002 and was developed by Protara Therapeutics.…

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Nipocalimab is Making Progress in Four Clinical Trials for Autoimmune and Alloimmune Conditions
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Nipocalimab is Making Progress in Four Clinical Trials for Autoimmune and Alloimmune Conditions

An investigative therapy for hemolytic disease of the fetus and newborn (HDFN) has just received the FDA's Rare Pediatric Disease and Orphan Drug Designations. This therapy is called nipocalimab and…

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New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress
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New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress

Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia…

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New Study Suggests Targeted Next Generation Sequencing Can Aid in Identification of Menkes Disease
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New Study Suggests Targeted Next Generation Sequencing Can Aid in Identification of Menkes Disease

  In a recent press release, biopharmaceutical company Fortress Biotech announced that their study regarding ATP7A targeted next generation DNA sequencing for Menkes disease was published in Molecular Genetics and Metabolism…

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Experimental Treatment for Recurrent Respiratory Papillomatosis Earns Orphan Drug Designation
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Experimental Treatment for Recurrent Respiratory Papillomatosis Earns Orphan Drug Designation

According to a story from PR Newswire, the biotechnology company INOVIO has just announced that the company's investigational product candidate INO-3107 has earned Orphan Drug designation from the US Food…

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