CAMBRIDGE -- Early-stage biotech companies are sharpening a new tool in their drug development workbench: patient advocacy.

Startups are tapping patients and patient groups to raise awareness of diseases, bankroll research, design clinical trials, and tell their stories to the regulators who decide which experimental medicines come to market.

“No one can doubt the power of the patient,’’ said Annie Ganot, the mother of a 6-year-old boy with Duchenne muscular dystrophy and head of patient advocacy at Solid Biosciences LLC, a Cambridge biotech developing a gene therapy treatment for the muscle-wasting disease. “The science should not be dictated by patients, but the patients’ voice should be heard.’’

Ganot and other speakers at a panel hosted Wednesday by the Massachusetts Biotechnology Council cited the recent successful campaign to persuade the Food and Drug Administration to green-light Exondys 51, a Duchenne drug developed by Cambridge’s Sarepta Therapeutics Inc.

Hundreds of patients and their families — including representatives of patient organizations that funded Sarepta’s clinical study — packed an FDA advisory committee meeting in April to offer emotional testimony in favor of the drug. The advisers and some FDA staffers contended the therapy had not been proved effective, but the agency overruled them and approved it.

Whether the Sarepta case will be a blueprint for other companies working on rare disease medicines remains to be seen. One panelist at Tuesday’s patient summit, at the Novartis Institutes for BioMedical Research in Cambridge, cautioned drug makers to keep some distance from patients when approaching regulators.

At meetings with regulators, “what we’re seeing is the FDA may slightly discount what patients are saying because industry brought them there,’’ said Alan Gilstrap, executive director of advocacy and policy at Akcea Therapeutics, a Cambridge company developing treatments for cardiometabolic lipid disorders. “So the best practice that we’ve learned is to let patient groups go on their own to tell their stories to the FDA and other regulatory agencies.’’

FDA officials are requiring Sarepta to conduct a new clinical trial — larger than its original 12-person study — to confirm the effectiveness of Exondys 51. The drug did not trigger harmful side effects, but experts differed on whether it produced enough of a key protein to slow the decline of muscle mass in Duchenne patients. Some critics argue that the FDA lowered its typical approval standards partly because of pressure from the patient advocates.

“You have to be careful about giving people false hope,’’ said Julie Kaufmann, a former primary care physician and retired medical director at Neighborhood Health Plan. “Patient advocacy can serve a purpose. Advocates pushed funding for AIDS research. But pushing funding is different from pushing approval’’ when the underlying science is uncertain.

Patient advocates said their role within companies setting prices for rare disease drugs is to urge striking a balance between letting the drug maker recoup its invest and fund future research and not creating a financial burden on patients and the health care system. Sarepta is charging an average of $300,000 per patient annually for Exondys 51, a price in line with those of treatments for other rare genetic disorders such as cystic fibrosis and Gaucher disease.

“The goal is to bring value to the patient and the health care system,’’ said Akcea’s Gilstrap.

However, The New York Times reported on Wednesday that some patient groups have been cautious on the issue of rising drug prices because they often get funding from drug makers.

Robert Weisman can be reached at robert.weisman@globe.com. Follow him on Twitter @GlobeRobW.