Rare Diseases News & Event
Important to know
08/06/2019 | EURORDIS
International rare disease events
July 9, 2019 | AJMC
Recommended
15 July 2019 | EURORDIS
Mar 28, 2019 | The Scientist
Researchers Analyze Epigenetic Signatures to Diagnose Rare Diseases
Mar 1 2019 | News Medical Life Sciences
New multi-year affiliation announced to establish global center for rare diseases
Feb. 20, 2019 | EURORDIS/Global Commision
Jan 8, 2019 | The Scientist
AI App Identifies Rare Genetic Disorders from Photos of Patients’ Faces
Updates
August 05, 2019 | AJMC
FDA Approves First Treatment for Symptomatic Tenosynovial Giant Cell Tumor
July 20, 2019 | AJMC
Report Shows How a t(15; 17) Aberration Typical of APL Was Really Myelofibrosis
July 5 2019 | AJMC
Burosumab Improves Symptom Relief in Genetic Rickets
May 25, 2019 | News Medical Life Sciences
Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert
May 24 2019 | FDA
May 23, 2019 | Rare Diseases International
Rare diseases feature for first time at World Health Assembly
May 2019 | NIH
New Method Could Help Find Potential Treatments for Rare Diseases
April 17 | AJMC
Cytomegalovirus May Speed Disease Progression in Cystic Fibrosis
April 10, 2019 | NIH
Releasing an immune system brake could help patients with rare but fatal brain infection
April 3, 2019 | NIH
FDA-approved drug effectively treats rare chronic immune disorder
Feb 20, 2019 | News Medical Life Sciences
BGU develops new AI platform for monitoring and predicting ALS progression
February 04 2019 | NIH
NIH's new automated toolset detects disease-causing genes in undiagnosed patients
August 13, 2018 | The Scientist
FDA Approves Oral Drug for Fabry Disease
Jun 20, 2018 | The Scientist
Positive Trial Results for Experimental DMD Gene Therapy