SINCE 1953 a number of reports seem to have established as a clinical entity a hereditary hemorrhagic disorder characterized by deficiency of antihemophilic globulin and a prolonged bleeding time.

Researchers have demonstrated for the first time that a new type of gene therapy, called RNA interference, can heal a genetic disorder in a live animal. RNA interference can "rescue" a strain of mouse ...

LONDON--(BUSINESS WIRE)--The rare genetic disease CDKL5 Deficiency Disorder (CDD) has been designated with a new disease code in the International Classification of Diseases (ICD), the medical ...

News-Medical speaks to Dr. Dan Lavery about the LouLou Foundation, a non-profit organization dedicated to CDLK5 Deficiency Disorder. Could you please tell us about the LouLou foundation? Why did the ...

Rian McCann is almost seven years old. Her parents, Danielle and Frank described her as kind, sassy, smart and above all, resilient. Rian was born with CDKL5 Deficiency Disorder — which prevents her ...

CDKL5 Deficiency Disorder (CDD) is a devastating neurodevelopmental disorder caused by mutations in the CDKL5 gene. The Loulou Foundation, a private non-profit foundation dedicated to the development ...

The rare genetic disease CDKL5 Deficiency Disorder (CDD) has been designated with a new disease code in the International Classification of Diseases (ICD), the medical classification list from the ...

Credit: Shutterstock. The application is supported by data from the phase 3 Marigold study. The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application for ...

Share on Facebook. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Full data have been published on ganaxolone (Ztalmy) for cyclin ...

A new study in mice finds that a gene therapy appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements and ...

Medically reviewed by Kierra Brown, RD Biotin deficiency can cause thinning hair and total loss of body hair. Pregnant people ...

I was inspired to write The Dance of Fear, because anxiety operates as such a powerful force in everyone's life, whether it holds us back in love and work-or propels us toward disaster. Indeed, ...