Rein Therapeutics ("Rein") , a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, ...

Patients with rare mitochondrial diseases already face a host of serious health issues, from muscle weakness to neurological problems and heart conditions.

Abeona Therapeutics' Zevaskyn, a cell therapy based on engineered keratinocytes harvested from patients, has been approved in the US as a treatment for the rare skin disorder recessive dystrophic ...

Sickle cell disease (SCD) affects an estimated 20 ... are reshaping clinical trial methodologies, particularly in the rare disease space. By streamlining data collection, enhancing patient ...

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