Trailblazing study discovers novel genetic causes of rare diseases, leading to improved diagnosis and better patient care July 6, 2020 by Gady Goldsobel
FDA Approves First Treatment for Adult Onset Still’s Disease, a Severe and Rare Disease June 17, 2020 by Gady Goldsobel
New test for rare disease identifies children who may benefit from simple supplement June 4, 2020 by Gady Goldsobel
New rare disease characterized by own facial features, cardiac defects and developmental delay May 19, 2020 by Gady Goldsobel
Researchers develop language test for people with Fragile X syndrome March 24, 2020 by Gady Goldsobel
How Gene Therapy May Hold Key to Treating Life-Threatening Cardiac Disease March 20, 2020 by Gady Goldsobel
A new therapeutic target against diseases caused by lipid accumulation in cells December 2, 2019 by Gady Goldsobel
Researchers discover potential new treatment for rare muscle-wasting disease November 21, 2019 by Gady Goldsobel
Scientists Identify Underlying Molecular Mechanisms of Alexander Disease November 21, 2019 by Gady Goldsobel
Promising Clinical Trial Results for Drug for Rare Disease in Which Patients Can’t Eat Fat August 8, 2019 by Gady Goldsobel
Researchers Uncover Pathways Leading to Heart Defects in Genetic Syndrome July 12, 2019 by Gady Goldsobel
Opitz C Syndrome: new advances to improve the genetic diagnose of an ultra-rare disease April 4, 2019 by Gady Goldsobel
Milk or no milk? Study fills long‑time knowledge gap on babies with genetic disorder March 19, 2019 by Gady Goldsobel
Muenzer Presents Additional Results from Groundbreaking Genome Editing Clinical Trial February 8, 2019 by Gady Goldsobel
First patient affected by a mutation in the nucleoside transporter SLC28 gene family February 5, 2019 by Gady Goldsobel
Genetic testing gives answers on developmental disorders during pregnancy February 1, 2019 by Gady Goldsobel
UC San Diego Researchers First to Use CRISPR/Cas9 to Control Genetic Inheritance in Mice January 24, 2019 by Gady Goldsobel
Experimental drug improves eye gaze behavior in fragile X syndrome January 19, 2019 by Gady Goldsobel
A study describes the dynamics of chromatin during organ and tissue regeneration December 4, 2018 by Gady Goldsobel
Can Genetic Therapy Help Kids with Angelman Syndrome Overcome Seizures? November 20, 2018 by Gady Goldsobel
Molecular site identified for targeted intervention of Fragile X syndrome November 8, 2018 by Gady Goldsobel
Zika biomarkers in blood could lead to prenatal screening for birth defects November 2, 2018 by Gady Goldsobel
New cell movement process key to understanding and repairing facial malformations October 22, 2018 by Gady Goldsobel
Study documents paternal transmission of epigenetic memory via sperm October 18, 2018 by Gady Goldsobel
Advances in Gene Editing and Delivery Offer Promise for People Living with Mitochondrial Diseases September 24, 2018 by Gady Goldsobel
