rare disease – Healthy USA

Sat. Mar 6th, 2021

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BioPharma biopharma nl Boston Daily Ensoma gene therapy Healthy Child Healthy Man Healthy Pets Healthy Women rare disease Startups SYN Top Story

How biotech startup Ensoma aims to take gene therapy beyond rare diseases

Feb 15, 2021 admin

Gene therapy may offer potential cures, but its promise comes with a price. Some experimental approaches require a multi-step…

Amicus Therapeutics BioPharma biopharma nl Cranbury Healthy Child Healthy Man Healthy Pets Healthy Women New Jersey Pharma Pompe disease rare disease SYN Top Story

Amicus’s path to unseat Sanofi in rare disease gets steeper after Phase 3 miss

Feb 12, 2021 admin

Amicus Therapeutics aims to position its experimental Pompe disease treatment as the new standard of care, supplanting a therapy…

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