Jan 28 (Reuters) - Regenxbio said on Wednesday the U.S. Food and Drug Administration has placed a clinical hold on two of its ...
Brelovitug has received breakthrough therapy designation from the US Food and Drug Administration.
Ashleigh McGeogh's daughter Delilah was diagnosed with Kawasaki disease, a condition that is the leading cause of acquired ...
In 2021, Jax was diagnosed with IPEX syndrome, an extremely rare condition that only impacts one in every 1.6 million people.
5don MSN
Woman with Rare Metabolic Disorder Adopts Girl with the Same Disease: 'She's My Mini-Me' (Exclusive)
Pam Kowalcyzk and her daughter have PKU, a rare disorder that prevents the body from processing protein ...
The son of an Arlington man who was detained by immigration agents last October has died, and his family is begging for the ...
Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for Mytesi® ...
Rare disease clinical trials are smaller and often use surrogate endpoints, reducing costs but posing unique operational ...
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...
When her newborn was diagnosed with Phenylketonuria (PKU), Jewelia Belcher tells PEOPLE it felt like her “world was crashing ...
The son of an Arlington man arrested by immigration officials late last year has died from a rare disease, prompting a fresh ...
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging ...
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