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Rare Diseases News & Event

Important to know

08/06/2019 | EURORDIS

International rare disease events

July 9, 2019 | AJMC

Upcoming EURORDIS events


15 July 2019 | EURORDIS

Rare disease patients support sharing of their health data while wanting to retain control of how and why data is used

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

Takeda, Microsoft and EURORDIS release report with action plan to help rare disease community shorten the diagnostic odyssey

Jan 8, 2019 | The Scientist

AI App Identifies Rare Genetic Disorders from Photos of Patients’ Faces


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

FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality

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

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