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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Companies Announce Collaboration to Develop Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

According to a story from globenewswire.com, the gene therapy company Fibrocell Science, Inc. and the biopharmaceutical company Castle Creek Pharmaceuticals have recently announced a collaboration between the two companies. The…

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Study Finds Firdapse is the Most Effective Treatment for LEMS, but it is Not The Most Commonly Prescribed

Results from the largest United States study to date of Lambert-Eaton myasthenic syndrome (LEMS) have indicated that for the majority of patients, Firdapse is the most effective treatment option currently…

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Edible Antibodies Halt E. Coli in Pigs, Could Help End Overuse of Antibiotics

According to a publication at BioPortfolio, Belgian researchers are developing an orally-administered antibody medication for patients living with gastrointestinal disorders. In most cases, antibodies are injected into the bloodstream for…

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Gene Therapy Displays Potential for Treating Charcot-Marie-Tooth Disease 4 Subtype C
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Gene Therapy Displays Potential for Treating Charcot-Marie-Tooth Disease 4 Subtype C

According to a story from Charcot-Marie-Tooth News, early preliminary testing of a possible gene therapy treatment for a certain type of Charcot-Marie-Tooth disease showed potential in a mouse model. This…

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Development of “Add-on” Therapy for Pulmonary Arterial Hypertension Ends After Poor Trial Showing
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Development of “Add-on” Therapy for Pulmonary Arterial Hypertension Ends After Poor Trial Showing

According to a story from Pulmonary Hypertension News, the drug developer United Therapeutics recently issued a statement in which the company announced that it was ceasing the development of esuberaprost…

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ISMRD International Conference for Glycoprotein Storage Diseases Coming This July

Upcoming Conference The International Society for Mannosidosis & Related Diseases (ISMRD) serves as an international patient advocate for those living with glycoprotein storage diseases. These include alpha-mannosidosis, fucosidosis, mucolipidosis type…

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Despite the Continued Appearance of Cases, Researchers Still Don’t Know Much About Acute Flaccid Myelitis
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Despite the Continued Appearance of Cases, Researchers Still Don’t Know Much About Acute Flaccid Myelitis

According to a story from AJC, three year old Reid Peteet is one of over 550 kids that have been diagnosed with acute flaccid myelitis since 2014. 2018 saw a…

Continue Reading Despite the Continued Appearance of Cases, Researchers Still Don’t Know Much About Acute Flaccid Myelitis