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Daily Archives: July 6, 2020

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Study on FMF Highlights Link Between Genomics and Disease
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Study on FMF Highlights Link Between Genomics and Disease

  • Post author:Jessica Lynn
  • Post published:July 6, 2020
  • Post category:Familial Mediterranean Fever

  Since the dawn of time, the human body has changed to cope with microbes and environmental changes. As a result, specific gene mutations evolved to protect populations from harm.…

Continue Reading Study on FMF Highlights Link Between Genomics and Disease
Givlaari Effects Sustained Over Time for Patients with Acute Hepatic Porphyria
whitesession / Pixabay

Givlaari Effects Sustained Over Time for Patients with Acute Hepatic Porphyria

  • Post author:Jessica Lynn
  • Post published:July 6, 2020
  • Post category:hepatic porphyria

  In 2019, Alnylam Pharmaceuticals received FDA approval for Givlaari, the first RNAi therapy for patients with acute hepatic porphyria. Although some were initially worried about the therapy's safety and…

Continue Reading Givlaari Effects Sustained Over Time for Patients with Acute Hepatic Porphyria
CBD Gel Could be Useful for Some Fragile X Syndrome Patients, Study Finds
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CBD Gel Could be Useful for Some Fragile X Syndrome Patients, Study Finds

  • Post author:James Moore
  • Post published:July 6, 2020
  • Post category:Fragile X Syndrome

According to a story from BioSpace, the drug company Zynerba Pharmaceuticals Inc. recently released the results from a clinical trial. This trial was testing the company's product Zygel™ (CBD gel)…

Continue Reading CBD Gel Could be Useful for Some Fragile X Syndrome Patients, Study Finds
David Fajgenbaum Found His Own Drug For Castleman’s Disease. Now He Turns to COVID-19
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David Fajgenbaum Found His Own Drug For Castleman’s Disease. Now He Turns to COVID-19

  • Post author:Sunniva Bean
  • Post published:July 6, 2020
  • Post category:Castleman's disease/COVID-19

David Fajgenbaum toes the line between patient and doctor. After growing up with a physician as a father, Fajgenbaum chose to follow in his footsteps. His most important lessons came…

Continue Reading David Fajgenbaum Found His Own Drug For Castleman’s Disease. Now He Turns to COVID-19
6-Year-Old with Scimitar Syndrome Underwent Open Heart Surgery

6-Year-Old with Scimitar Syndrome Underwent Open Heart Surgery

  • Post author:Jessica Lynn
  • Post published:July 6, 2020
  • Post category:Rare Disease/Scimitar syndrome

In 2016, 6-year-old Alec Ahmad loved club soccer, playing games with his two brothers, and running around with his friends at recess. He was full of energy. But every once…

Continue Reading 6-Year-Old with Scimitar Syndrome Underwent Open Heart Surgery
Restrictions Eased on Cannabinoid Drug That Reduces Childhood Seizures

Restrictions Eased on Cannabinoid Drug That Reduces Childhood Seizures

  • Post author:Sunniva Bean
  • Post published:July 6, 2020
  • Post category:Epilespy/Rare Disease

A cannabinoid medication that uses a non-psychoactive ingredient of cannabis to treat rare forms of epilepsy has now been recategorized as a schedule 5 drug in the UK, the least…

Continue Reading Restrictions Eased on Cannabinoid Drug That Reduces Childhood Seizures
2 Patients with XLMTM Die in ASPIRO Trial
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2 Patients with XLMTM Die in ASPIRO Trial

  • Post author:Jessica Lynn
  • Post published:July 6, 2020
  • Post category:Hemophilia/Rare Disease/Timely

At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…

Continue Reading 2 Patients with XLMTM Die in ASPIRO Trial
Raise Your Resilience: Standing Strong through COVID-19
Source: Pixabay

Raise Your Resilience: Standing Strong through COVID-19

  • Post author:Jessica Lynn
  • Post published:July 6, 2020
  • Post category:COVID-19/Mood Disorder

If you're experiencing a difficult time with COVID-19, you're not alone. Many people, including myself, are struggling with managing anxiety, stress, and isolation. Additionally, this time period can feel traumatic,…

Continue Reading Raise Your Resilience: Standing Strong through COVID-19
New Methods To Evaluate Stargardt Disease Treatments
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New Methods To Evaluate Stargardt Disease Treatments

  • Post author:Kendall Mason
  • Post published:July 6, 2020
  • Post category:Stargardt Disease

Vision loss is a major symptom of Stargardt disease, so a main goal of treatments is to combat this loss. In order to test the effectiveness of these treatments, there…

Continue Reading New Methods To Evaluate Stargardt Disease Treatments

Featured


Picture of Family


Metastatic Breast Cancer: Navigating Grief


Picture of Ralph Family walking


Rethinking What It Means to Live With Acromegaly


Illustration of mentor program members


The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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