Myrtelle Inc. has just announced that they have been granted three new designations from the FDA for their investigative gene therapy called rAAV-Olig001-ASPA. This therapy is being investigated as a…
Myrtelle, via Business Wire News, reports the completion of Phase 1/2 investigating its gene therapy for Canavan disease (CD) which they have now expanded to treat younger patients. Thus far,…
Canavan disease is an ultra-rare condition that currently has no FDA-approved therapies, meaning that patients face a severe unmet medical need. BridgeBio Pharma aims to change this with their investigational…
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According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…
Gene therapies have huge potential in the field of rare disease. The fact that these therapies could offer a potential cure otherwise fatal diseases has given families incredible hope. Unfortunately,…
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
Canavan disease is a type of leukodystrophy frequently found in the Ashkenazi Jewish population. It's a rare genetic condition which is ultimately fatal. It's all caused by a deficiency of the aspartoacylase…
Cedar Rapids, Iowa is rallying to help a one-year-old girl who has been diagnosed with Caravan disease, a devastating disorder that often leads to premature death, within mid-childhood. What is Canavan…
Two children from a Brooklyn family were born with an ultra rare genetic disease called Canavan disease. Their parents' research revealed that gene therapy could possibly treat the disease. Unfortunately,…
At six years old, Benny was unable to sit up, roll over and scoot like other babies his age. Missing all the major milestones, his mother Jennie knew that something…
Canavan disease is an isolating condition. It's a rare disorder caused by a genetic mutation that can be passed on in families. It damages nerve cells, so they're not able…
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