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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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