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Rare Diseases in Science

Recommended

Apr. 17, 2019 | NORD

The 2019 Rare Neurological Disease Special Report Is Now Available


December 04, 2018 | MD Magazine

Jasmine Zain, MD, Highlights Current & Upcoming T-Cell Lymphoma Treatments


 

Updates


Jul 25 2019 | News Medical Life Sciences

Novel therapeutic strategy for treating Lafora Disease shows promise in preclinical study


Jul 25 2019 | News Medical Life Sciences

Genetic mutations link rare skin disorder and non-alcoholic fatty liver disease


July 20, 2019 | AJMC

Report Shows How a t(15; 17) Aberration Typical of APL Was Really Myelofibrosis


Jun. 25, 2019 | NORD

NORD Awards Ten New Grants in Eight Disease States for Rare Disease Research


June 20, 2019 | AJMC

Adding Hydroxyurea to Ruxolitinib Yields Higher Clinical Response in Myelofibrosis


June 18, 2019 | AJMC

Is Leukocytosis a Risk Factor for Thrombosis in Patients With Myeloproliferative Neoplasms?


Jun 18 2019 | News Medical Life Sciences

Study uncovers potential modifier genes in patients with Charcot-Marie-Tooth disease


May 25 2019 | News Medical Life Sciences

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert


May 2 2019 | News Medical Life Sciences

CU Anschutz researchers identify new immunodeficiency disease


May 2 2019 | News Medical Life Sciences

Scientists discover first gene involved in lower urinary tract obstruction


April 27, 2019 | AJMC

Ruxolitinib Resolves Sarcoidosis in Patient With PV


April 20, 2019 | AJMC

Splenectomy Before Stem Cell Transplant May Help Certain Patients With MF


Apr. 16, 2019 | NORD

NORD Publishes Report on Primary Distal Renal Tubular Acidosis


Apr 8 2019 | News Medical Life Sciences

Study demonstrates viability of gene editing strategy for recessive dystrophic epidermolysis bullosa


April 3, 2019 | NIH

FDA-approved drug effectively treats rare chronic immune disorder


Mar 28, 2019 | The Scientist

Researchers Analyze Epigenetic Signatures to Diagnose Rare Diseases


Mar 25 2019 | News Medical Life Sciences

Inherited form of rickets improves more with new injectable medicine than conventional therapy


Mar 20 2019 | News Medical Life Sciences

Royal Holloway professors to lead new to research into curing Neurofibromatosis type 1


Nov. 29, 2018 | NORD

NORD Publishes Report on Post-Transplant Lymphoproliferative Disease


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