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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Striking a Balanced View of Clinical Trials
This month marks 13 years since my sister’s diagnosis of CLN1 disease, or Batten disease. That means I’ve had 4,745 days — or 113,880 ... Read more -
Brain-Directed Gene Therapy May Prevent Neurodegeneration in CLN6 Disease, Study Says
Brain-directed gene therapy is a feasible therapeutic strategy to prevent neurodegeneration associated with CLN6 disease, a mouse study says. The findings of 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 -
Motor Impairments Are Early Sign of Juvenile Batten Disease, Study Finds
In most patients with juvenile Batten disease, motor impairments are already apparent at the time of diagnosis and are not related to vision loss, ... Read more
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3.1 Ways to Fight Your Rare Disease
My life changed forever 13 years ago. In the space of a few months, ... -
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 ... -
FDA Grants Fast Track Designation to ABO-202 Gene Therapy for Infantile Batten Disease
Abeona Therapeutics’ one-time gene therapy candidate ABO-202 has been granted fast track designation by the U.S. Food and Drug ... -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
Fycompa May Help Control Involuntary Muscle Jerks in Patients With CLN2 Disease, According to Case Report
Fycompa (perampanel) may be considered as an add-on therapy to control myoclonus (quick, involuntary ...
North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs
News
A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free programs to help children with Batten disease and other special needs build self-esteem. Since launching these programs, Indo Jax has been featured ... Read more