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5 advancements in rare disease research over the past year

10 biotech companies making a difference in rare diseases

Beyond Biotech podcast 35: Rare Disease Day

Moderna and Life Edit Therapeutics look to speed up gene editing therapies

First patient enrolled in Pharming APSD trial  

FDA approves alpha-mannosidosis drug

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Positive results for INOVIO recurrent respiratory papillomatosis trial

Janssen reports positive topline phase 2 HDFN results

Hundreds of genetic discoveries from FinnGen study 

In Depth 11 Jan 2023

Drug repurposing emerges as viable option for rare disease treatment

Opinion 12 Dec 2022

Artificial intelligence: a new era in rare genetic disease diagnosis

PacBio and Boston Children’s Hospital to study genome to benefit patients

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