Onlymyhealth20h
What Is Sanfilippo Syndrome: Symptoms, Treatment, and the Impact on Children’s Lives
Discover Sanfilippo Syndrome a rare genetic disorder affecting childrens development Learn about its symptoms treatment ...
pharmaphorum23h
First rare disease therapies chosen for FDA START programme
Denali’s candidate is its DNL12 enzyme replacement therapy (ERT) for mucopolysaccharidosis ... Neurogene has contributed its gene therapy NGN-401 for Rett syndrome, a rare genetic neurological ...
Cystic Fibrosis Foundation17h
About Cystic Fibrosis
Learn the facts on our page, Dispelling Misconceptions About Cystic Fibrosis. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein ...