April 30th is the First-Ever AMKD Awareness Day. Here’s What You Need to Know
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April 30th is the First-Ever AMKD Awareness Day. Here’s What You Need to Know

  On April 30, 2024, the American Kidney Fund (AKF) will be organizing the first annual APOL1-mediated kidney disease (AMKD) Awareness Day. This awareness day will be a time to…

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Clinical Trial: Rinvoq 45 mg Brings ‘Rapid Resolution’ of Crohn’s Disease Symptoms
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Clinical Trial: Rinvoq 45 mg Brings ‘Rapid Resolution’ of Crohn’s Disease Symptoms

Patients with moderate to severe Crohn’s disease have found quick relief from abdominal pain and stool frequency during the first week of induction therapy according to data analysis published in…

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Investigational Treatment for ADPKD Earns FDA’s Orphan Drug Designation
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Investigational Treatment for ADPKD Earns FDA’s Orphan Drug Designation

Approximately 540,000 individuals in the United States have been diagnosed with autosomal dominant polycystic kidney disease (ADPKD). The disease causes persistent and rapidly accumulating cysts to grow in the patient’s…

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Is the CDC Botching its RSV Vaccine Rollout?
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Is the CDC Botching its RSV Vaccine Rollout?

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Last fall, a new vaccine for respiratory syncytial virus (RSV) intended for pregnant people was made available to the public. Physician Sarah Turner, who was used to getting some pushback…

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Rare Community Profiles: How a Clinical Study Changed Dana’s Trajectory After Desmoid Tumor Recurrence
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Rare Community Profiles: How a Clinical Study Changed Dana’s Trajectory After Desmoid Tumor Recurrence

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

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Scientists have Discovered a Connection Between Metabolic Problems in the Brain and Neurological Disorders
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Scientists have Discovered a Connection Between Metabolic Problems in the Brain and Neurological Disorders

  These efforts led to collaboration by the International Brain pH Project that involves 131 scientists in 105 labs from seven countries. The scientists have identified changes in lactate levels…

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Rare Community Profiles: How Kenzi is Navigating the Challenges of MCTD, FCAS, and Medical PTSD
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Rare Community Profiles: How Kenzi is Navigating the Challenges of MCTD, FCAS, and Medical PTSD

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

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Science Simplified: Differences Between a Gene Therapy Clinical Trial and a Cure
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Science Simplified: Differences Between a Gene Therapy Clinical Trial and a Cure

Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…

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Researchers Discover ‘Super Minigene’ that Helps Find Potential Therapies for Spinal Muscular Atrophy
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Researchers Discover ‘Super Minigene’ that Helps Find Potential Therapies for Spinal Muscular Atrophy

For decades researchers have had to isolate segments of genes in order to study them. They called the isolated gene a “mini gene.” Ravindra Singh, a biomedical science professor at…

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Gene Therapy Led to “Robust Microdystrophin Expression” in Duchenne Muscular Dystrophy Patient
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Gene Therapy Led to “Robust Microdystrophin Expression” in Duchenne Muscular Dystrophy Patient

Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…

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