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