Proteostasis Therapeutics received orphan drug designation from the European Commission for its investigational CFTR amplifier PTI-428 in…
Articles by Alice Melão, MSc
Rapid weight gain early in life may have a long-term negative impact on the growth of children with cystic…
Treatment with Proteostasis Therapeutics’ triple combo therapy regimen may significantly improve the lung function of patients with cystic…
Targeting some elements in the bacterial cell wall can be an effective way to activate the immune system and fight…
Vertex Pharmaceuticals is planning to request regulatory approval from the U.S. Food and Drug Administration (FDA) and the European Medicines…
An ongoing Phase 1/2 clinical trial exploring Translate Bio’s investigational therapy MRT5005 will start testing multiple ascending doses…
Eloxx Pharmaceuticals will explore the potential of its lead investigational drug candidate, ELX-02, as a treatment for patients…
A new strategy to genetically prevent disease-related mutations from occurring shows the potential to treat several genetic diseases, including…
Results from a Phase 3 trial demonstrate that Symkevi (tezacaftor and ivacaftor combo), known as Symdeko in…
The American Thoracic Society (ATS) recommends the use of home oxygen therapy in children with cystic fibrosis (CF)…