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 ...
Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for ...
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 ...
A drug being developed by Boehringer Ingelheim for COVID-19 and reinvented as a potential therapy for kidney disease – and has now hit the mark in a phase 2 trial. TRPC6 inhibitor apecotrep (BI 764198 ...
Researchers at UCL and Great Ormond Street Hospital (GOSH) have developed the first-ever lab-grown mini-stomach that contains ...
Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...
Backed with $82 million and led by two of BioMarin’s former executives, Mendra plans to acquire rare disease assets and use ...
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
NORD's growing network, now 46 institutions strong, represents a coordinated nationwide effort to strengthen rare disease care and research collaboration With these new members, the network now ...
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