The research at Bristol Children's Hospital into Duchenne muscular dystrophy (DMD) is being funded by charity Action Duchenne.
Paediatric consultant Dr Anirban Majumdar, said it was "very exciting" and the drugs being tested could be a "potential cure".
DMD is a severe progressive disease and mostly affects boys.
Brothers Aaron and Jack Ebanks, 19 and 16, from Birmingham, who have the condition, are raising money and awareness of the disease by travelling from Bristol to Birmingham by wheelchair. They set off on Friday and hope to arrive on Monday.
Jack said they wanted "to prove to people anything's possible if you're disabled" and "having Duchenne shouldn't stop you doing anything in life".
Dr Anirban Majumdar, from Bristol Children's Hospital, said: "This is a very exciting time for boys with DMD, because for the first time in many many years we now have a number of research projects that are up and running in the country.
"And we here in Bristol are able to start delivering some of this research. It's very important because for the first time there are a number of drugs that are being tested as a potential cure and disease-modifying treatment for these boys."
Diana Ribeiro, from Action Duchenne, said: "We've been working with Bristol for a while. We firmly believe that all patients living with Duchenne should have access to trials and clinics across the UK."
A clinical trial into treatment for children with a potentially fatal neuromuscular disorder has begun.