Press Release: Rare Disease Community Calls on Congress & FDA to Enact Life-Saving Public Policy Solutions

Contact: Britta Vander Linden bvanderlinden@everylifefoundation.org 917.604.6518 30 million Americans need more treatment and diagnostic opportunities https://everylifefoundation.org/rare-disease-community-calls-on-congress-fda-to-enact-life-saving-public-policy-solutions/ (Washington, D.C., December 4, 2019) Hundreds of rare disease advocates from around the country were brought to Washington,…

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ICYMI: #PatientsAreNotFaking Because Patients Are Sick, And They Are Sick of Not Being Taken Seriously
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ICYMI: #PatientsAreNotFaking Because Patients Are Sick, And They Are Sick of Not Being Taken Seriously

Getting a diagnosis for a rare disease is hard. Not many people are familiar with the many obscure symptoms and manifestations of these diseases. Because of this, patients with rare…

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After Five Insurance Coverage Denials, the Next Appeal Brought a Young Patient Closer to Receiving Treatment for Metachromatic Leukodystrophy

  A recent article in the Taunton Gazette describes the dilemma faced by the parents of four-year-old Jaxtien Miller who is a metachromatic leukodystrophy patient waiting for a stem cell…

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IYCMI: CALQUENCE Has Received US FDA Approval to Benefit Adults with CLL
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IYCMI: CALQUENCE Has Received US FDA Approval to Benefit Adults with CLL

  A recent article in BioPortfolio carried AstraZeneca’s announcement of the FDA’s approval of CALQUENCE® (acalabrutinib) for adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The approval…

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Study Finds Increased Risk of Bladder Problems for Primary Sjögren’s Syndrome Patients
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Study Finds Increased Risk of Bladder Problems for Primary Sjögren’s Syndrome Patients

A recent study published in the scientific journal PLOS One has determined that patients with the rare disorder primary Sjögren's syndrome are at a greater risk of bladder problems than previously thought.…

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CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 
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CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 

   CRISPR/Cas9 technology reached another milestone with encouraging results in two recent trials of CTX001. An article in Pharmaceutical Technology carried an announcement by CRISPR Therapeutics and Vertex Pharmaceuticals about…

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