There are more than 10,000 individual rare diseases and most are without therapy.
Personalized genetic therapy represents one promising approach for their treatment. We …

Charcot-Marie-Tooth disease (CMT), the most common hereditary neuropathy, currently
lacks an FDA/EMA-approved drug, and its management still relies on rehabilitation therapy …

The remarkable efficacy of recombinant adeno-associated virus gene therapy for rare
genetic diseases, such as spinal muscular atrophy type 1 (SMA1), 1 has raised expectations …

Beta-propeller protein-associated neurodegeneration (BPAN) is an ultra-rare, X-linked
dominant, neurodegenerative disease caused by loss-of-function mutations in the WDR45 …

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کتابخانه آنلاین زبان اصلی کتابخانه آنلاین فارسی ویدیوی آموزشی نرم افزار مطب و کلینیک نوبت …‎