The National Organization for Rare Disorders (NORD), is a unique federation of voluntary health organizations dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.

EURORDIS is a non-governmental patient-driven alliance of patient organisations and individuals active in the field of rare diseases, dedicated to improving the quality of life of all people living with rare diseases in Europe.

NIH (National Institutes of Health) -

Hide & Seek Foundation -
Hide & Seek is a community of people dedicated to finding treatments and cures for a devastating genetic condition called Lysosomal Disease.

The Childhood Liver Disease Research and Education Network -

The Childhood Liver Disease Research and Education Network (ChiLDREN) is a collaborative team of doctors, nurses, research coordinators, medical facilities and patient support organizations. The ChiLDREN Network has clinical sites and research labs in the US and Canada, and also includes a research lab in London. These sites are working together to improve the lives of children and families dealing with rare liver diseases.

Children’s Rare Disease Network -
The Children’s Rare Disease Network will create greater public awareness for rare disease, while connecting, educating and empowering the millions of families and caregivers affected, through an online community and collaborative portal.

Synageva BioPharma Corp is Now Alexion. -
Synageva BioPharma was a biopharmaceutical company dedicated to discovering, developing and delivering medicines for patients with rare diseases and unmet medical need. Synageva developed a potential enzyme replacement therapy for LAL D.  It is currently in clinical trials to evaluate the safety and efficacy in infants, children and adults. Synageva was bought by Alexion in 2015. Alexion continues the tradition of developing treatments for rare and ultra rare genetic conditions. Enzyme replacement therapy  studies for LAL-D are unchanged, and ERT is in development through the same clinical trials, now sponsored by Alexion.

Genetics Home Reference -
This is the National Library of Medicine that provides consumer oriented information about genetic conditions, including a Help Me Understand Genetics Handbook.


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