European Initiative Targets Diagnosis, Treatment of Rare Diseases
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while ... Read more
Updates
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Measures of Quality of Life Used for Children with Rett Also Valuable During Adulthood, Study Suggests
Different domains used to assess quality of life in children with Rett syndrome also may be useful for adults, a study suggests. The study, ... Read more -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing ... Read more -
First Patient Dosed in Phase 2 Extension Study of Anavex 2-73
An extension study of a Phase 2 trial investigating Rett syndrome therapy candidate Anavex 2-73 has dosed its first patient. “We are delighted to ... Read more
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Subtle Signs of Rett Could Be Opportunity for Early Intervention, Study Says
Subtle but apparent signs of disease may precede the onset of overt symptoms in ... -
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy ... -
L-serine Improved Outcomes in Girl with Atypical Rett Syndrome, Case Report Says
Dietary supplementation with the amino acid L-serine improved motor and communication skills in a ... -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
Phase 2 AVATAR Study Testing Anavex 2–73 for Rett Syndrome Doses First Patient in Australia
The first patient has been dosed in Anavex Life Sciences’ AVATAR Phase 2 trial, taking ...
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