Safe Pregnancies with Rheumatic Diseases are Possible with Planning
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Safe Pregnancies with Rheumatic Diseases are Possible with Planning

Dr. Lisa Sammaritano from Weill Cornell Medical College has explained a way to lower risks within pregnancy for those with rheumatic diseases. Pregnancy and Rheumatic Diseases Patients who have a…

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Results from Two Studies Show Soticlestat Efficacy for CDD and Dup15q Syndrome
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Results from Two Studies Show Soticlestat Efficacy for CDD and Dup15q Syndrome

  Developmental and epileptic encephalopathies (DEEs), or rare epilepsies, are somewhat of a newer topic. However, researchers are already making strides in creating treatments to address unmet patient needs. On…

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The TARGET-NASH Study Finds Prior Opioid Use Prevalent in NAFLD Patients
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The TARGET-NASH Study Finds Prior Opioid Use Prevalent in NAFLD Patients

Target RWE, an innovative health evidence solutions company based in Durham, North Carolina recently announced its current data from the study of nonalcoholic steatohepatitis (NASH) via PRNewswire. The study, entitled…

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A New Mutation Causing Atypical Hemolytic Uremic Syndrome Has Been Discovered
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A New Mutation Causing Atypical Hemolytic Uremic Syndrome Has Been Discovered

Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…

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The First Patient has Been Dosed with an Investigative Therapy for Acute Myeloid Leukemia

Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…

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Possible XSCID Treatment MB-207 Granted Orphan Drug Designation
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Possible XSCID Treatment MB-207 Granted Orphan Drug Designation

  Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…

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EC Approves Marketing Authorization of AYVAKYT for Patients with PDGFRA D842V Mutant GIST
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EC Approves Marketing Authorization of AYVAKYT for Patients with PDGFRA D842V Mutant GIST

  Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…

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MN-166 (Ibudilast) Shows Promise for CIPN
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MN-166 (Ibudilast) Shows Promise for CIPN

  Early last week, biopharmaceutical company MediciNova Inc. ("MediciNova") announced promising results for their therapeutic candidate MN-166 (ibudilast). The company, which published in the findings in Cancer Chemotherapy and Pharmacology, explored…

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