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Daily Archives: May 28, 2020

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Patients Report Effects of Tolvaptan on ADPKD
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Patients Report Effects of Tolvaptan on ADPKD

  • Post author:Patient Worthy Contributor
  • Post published:May 28, 2020
  • Post category:Autosomal Dominant Polycystic Kidney Disease

By Danielle Bradshaw from In The Cloud Copy Researchers in Germany have gone on record saying that there is a concern with how well patients are able to tolerate the…

Continue Reading Patients Report Effects of Tolvaptan on ADPKD
Gender Plays Role in Motor Symptoms of Lab Rats with Cerebral Palsy
source: pixabay.com

Gender Plays Role in Motor Symptoms of Lab Rats with Cerebral Palsy

  • Post author:Patient Worthy Contributor
  • Post published:May 28, 2020
  • Post category:Cerebral Palsy/Rare Disease

By Jodee Redmond from In The Cloud Copy The results of a new study using laboratory rats to model cerebral palsy with spasticity (muscle stiffness) accompanied by dystonia (involuntary muscle…

Continue Reading Gender Plays Role in Motor Symptoms of Lab Rats with Cerebral Palsy
New Clinical Expectations Needed to Treat Hemophilia
Photo by National Cancer Institute on Unsplash

New Clinical Expectations Needed to Treat Hemophilia

  • Post author:Jessica Lynn
  • Post published:May 28, 2020
  • Post category:Hemophilia/Hemophilia A

The medical realm is changing - but how does that impact patient care and clinical expectations? The National Hemophilia Foundation discusses how clinical expectations for patients with hemophilia don't necessarily…

Continue Reading New Clinical Expectations Needed to Treat Hemophilia
A SF3B1 Mutation May Cause A New Subset of Myelodysplastic Syndromes
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A SF3B1 Mutation May Cause A New Subset of Myelodysplastic Syndromes

  • Post author:Jessica Lynn
  • Post published:May 28, 2020
  • Post category:Myelodysplastic syndromes/Rare Disease

Genetic research is a burgeoning and exciting field. With the discovery of new genetic mutations and variants comes a deeper understanding of rare diseases and their subtypes. According to Cancer…

Continue Reading A SF3B1 Mutation May Cause A New Subset of Myelodysplastic Syndromes
A Conversation With a Multiple Sclerosis Patient and Her Husband

A Conversation With a Multiple Sclerosis Patient and Her Husband

  • Post author:Sunniva Bean
  • Post published:May 28, 2020
  • Post category:Multiple Sclerosis/Rare Disease

As reported in Multiple Sclerosis News Today, Jessie Ace tells the story of receiving her diagnosis with multiple sclerosis at 22. She wrote, "I hate needles." She ranks being told…

Continue Reading A Conversation With a Multiple Sclerosis Patient and Her Husband
Mouse Model Provides Insight into MEF2C Haploinsufficiency Syndrome

Mouse Model Provides Insight into MEF2C Haploinsufficiency Syndrome

  • Post author:Trudy Horsting
  • Post published:May 28, 2020
  • Post category:Fragile X Syndrome/Rare Disease

Christopher Cowan and his lab at the Medical University of South Carolina recently conducted a study in mice in order to understand how defective MEF2C genes impact brain development. A…

Continue Reading Mouse Model Provides Insight into MEF2C Haploinsufficiency Syndrome
Study: Rituximab as a Treatment for Myasthenia Gravis
qimono / Pixabay

Study: Rituximab as a Treatment for Myasthenia Gravis

  • Post author:Kendall Mason
  • Post published:May 28, 2020
  • Post category:Myasthenia Gravis

A study has recently been conducted that suggests that rituximab may be preferable over traditional treatments for myasthenia gravis. It also concluded that tiuximab is the most effective if it…

Continue Reading Study: Rituximab as a Treatment for Myasthenia Gravis
How NORD is Supporting Rare Disease Patients During this Pandemic

How NORD is Supporting Rare Disease Patients During this Pandemic

  • Post author:Trudy Horsting
  • Post published:May 28, 2020
  • Post category:COVID-19/Rare Disease

NORD has received thousands of phone calls and emails each week from concerned rare disease patients. They are asked for advice and how to decipher the myriad of contradictory information…

Continue Reading How NORD is Supporting Rare Disease Patients During this Pandemic
Gene Therapy for Pompe Disease has Produced Positive Outcomes in Mouse Models

Gene Therapy for Pompe Disease has Produced Positive Outcomes in Mouse Models

  • Post author:Trudy Horsting
  • Post published:May 28, 2020
  • Post category:Pompe Disease

AVROBIO AVROBIO is based out of Cambridge, Massachusetts. They specialize in single-dose gene therapy as a cure for rare genetic conditions such as cystinosis, Gaucher disease, and Fabry disease. Most…

Continue Reading Gene Therapy for Pompe Disease has Produced Positive Outcomes in Mouse Models
COVID-19 Antibody Tests: Are They Oversold?

COVID-19 Antibody Tests: Are They Oversold?

  • Post author:Rose Duesterwald
  • Post published:May 28, 2020
  • Post category:COVID-19

According to a recent article in Nature, many countries are anticipating an immediate response from newly developed antibody tests. These tests are slated to bring relief from the lockdown and…

Continue Reading COVID-19 Antibody Tests: Are They Oversold?
Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation
qimono / Pixabay

Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation

  • Post author:Kendall Mason
  • Post published:May 28, 2020
  • Post category:Sanfilippo Syndrome

Seelos Therapeutics has recently received the Rare Pediatric Disease Designation for their Sanfilippo syndrome treatment, trehalose. This designation is an incentive for companies to make therapies for conditions with an…

Continue Reading Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation

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