"Max Rowan and Charlie Vertins muscles have been slowly collecting damage since the day they were born. Someday they will not be able to walk. They will not be able to stand. Eventually they will not be able to live. The brothers ages  through  suffer from a debilitating and deadly genetic condition called Duchenne muscular dystrophy DMD. Yet on September  Max Rowan and Charlie Vertin got dressed up. In dark suits and blue shirts with serious expressions on their round faces they walked to stand next to their mother in a very beige room outside Washington D.C. The people in that room they believed could change the course of their lives. For years the Vertin brothers have been participating in a clinical trial for a new medication called ataluren intended to keep the progression of their disease at bay. By October  theyll know if they will be able to keep taking that drug. That is the deadline for the Food and Drug Administration to make a choice. The agency could approve the drug allowing the brothers and others like them to continue to take it. Or it could reject the drug for the third time and throw them into an uncertain future. An FDA committee has already voted that data about the drugs effectiveness was inconclusive. The agencys final decision almost always echos the decisions of its committees. So if this were any other kind of drug at any other time in FDA history the FDAs final answer would be a very predictable no. If they do say no the Vertins could lose access to the drug that has stabilized their condition. But theres still a chance the FDA could approve the drug. Input from people affected by DMD may have convinced the agency once before to approve a drug for the condition despite iffy data. That is what the Vertinsand the company that produces the drugare hoping will happen again. Falling Behind It started out so innocent his mother Betty Vertin told Newsweek. Max was a big infant born  lbs  oz and nearly two feet long. When he was missing milestones I just thought it was because it was a big baby. Even doctors assumed that he would eventually catch up. But when he started preschool the difference between Max and other children became more obvious. When other kids were running and jumping and climbing Max wasnt doing those things. He was just behind physically. When Max was  in  a genetic test confirmed that he had DMD. Betty had never heard of it. Before we started living this life I was clueless she said.  DMD is one of a class of similar genetic disorders. About  boys out of every  in the United States under  years old have DMD or a milder form called Becker muscular dystrophy. Most of the children affected are boys. The illness affects the way their legs heart and lungs work. Maxs diagnosis was devastating for Betty. I feel like I walked into the room with a healthy fouryearold boy and I walked out with a child that was dying she said. But the familys struggles with DMD were just beginning. At the time Rowan was  and Betty suspected that he also had the disease shed seen how her son struggled to stand up onto a stepstool in the bathroom to brush his teeth. She was also ten weeks pregnant with Charlie. Genetic tests eventually confirmed both Rowan and Charlie also had DMD. Bettys two daughters Lexi  and Mary  as well as a fourth son Chance  do not. I felt like we had a year and a half  of the same horrible diagnosis over and over and over again she said. It was the hardest  months Ive ever had and I hope I ever had. Still Walking DMD always results in muscle weakness due to a lack of a protein called dystrophin. Some of the newest treatments for the condition try to fix that. One drug which the FDA approved in  works for people whose illness springs from a mutation in the gene that codes for dystrophin. The drug called Exondys  or eteplirsen allows the genetic machinery that produces the protein to skip over the mutated part to make something that will work better. About  percent of people with DMD have this particular mutation according to https the FDA. The Vertin brothers are not among them the drug shouldnt help them at all. So they tried ataluren. The drugs brand name is Translarna. The company that produces ataluren PTC Therapeutics says that its drug forces cells to make the proteins in spite of a different kind of mutation called a nonsense mutation. Instead of having an error in the protein the protein is accidentally cut short. This is the kind of mutation that three of Betty Vertins children have. The Vertins have received the drug free through the clinical trial and expenses for their trip from Nebraska to Washington D.C. were paid by PTC Therapeutics.  Ataluren works by ordering a cell to use a slightly different piece to build the proteinas if someone spraypainted the word dont on the molecular version of a stop sign. Exactly how it does that is still mysterious. A paper published by the companys scientists in the Proceedings of the National Academies of Science http said it probably affected the ribosome a key part of how proteins are put together in a cell. Researchers not affiliated with the company did not find httpjournals.plos.orgplosbiologyarticleid.journal.pbio. the same kind of activity. Regardless of what mutation causes the illness most people with DMD die in their s. Betty was told to expect her oldest son Max to be in a wheelchair fulltime by the time he was  or . Max is  now. His th birthday is in early November. He uses an electric mobility scooter for long distances but he can still walk. He rides a bike without training wheels and without any modifications. He has a part in a school play and goes up and down stairs to get to the stage. He even plays a trumpet in his schools band. Hes got the lung capacity to blow that thing very loudly Betty said. For that Betty credits ataluren which Max started three years ago. His life isits not like his peers but hes getting a lot of the experiences that his peers are having she said. Hes living a sort of a typical sixthgrade life and that diagnosisI didnt know what it would look like for him. I didnt expect it to look like this. But not everyone considers ataluren to be a miracle drugwith good reason. The clinical trial results havent been great. Most of the companys studies tested how fast a child could go about  feet or walk up and down stairs or how far they could walk in six minutes. They did see some slight positive results many of which came from a study done with the specific kind of patient the company discovered benefited most in a previous trial. The FDA needs good data to make their decision because when the agency approves a drug is sends a signal to the world. An approval is the FDA saying this drug is safe and effective. You could use it for your child too. PTC Therapeutics has tried to get FDA approval for the drug twice before. Both times the agency refused to even consider the application. This time the application for approval was filed over the FDAs protest. The drug has been conditionally approved in Europe. A Million Dollar Issue Part of the reason that the drug has struggled is because of this requirement if the drug is effective the data should show it. But that hasnt happened consistently with ataluren. Many of the clinical trials failed to satisfy the statistical requirement to prove a drug worksat least not for the main outcome the company was looking at. Other secondary measurements did seem to have good statistics. Certain groups of children seemed to have better results than others too. PTC Therapeutics founder and CEO Stuart Peltz says the statistics arent a reason to reject the drug. Just because a drug doesnt measure up in that way he says doesnt mean the drug is not effective.  But even if the FDA agrees to overlook some of the negative data about ataluren insurers may not. That could be a milliondollar issue for the families with affected children. If the evidence isnt good then insurance companies wont pay for drugs said National Center for Health Research NCHR president Diana Zuckerman. Two recently approved drugs have run into this problem Spinraza a drug for spinal muscular atrophy and Exondys . Both drugs cost hundreds of thousands of dollars a year. Pharmaceutical companies traditionally will not discuss prices before drugs are approved that said ataluren costs about  in Britain https During clinical trials participants like the Vertins normally do not pay for the treatment. The NCHR also presented at the FDA meeting in September to discuss ataluren urging the agency to not approve the drug http without more research. The organizations statement also raised concerns about side effects associated with the drug specifically about the chance that it raises blood pressure and cholesterol levels in children. The company says these shifts are small and not clinically significant. In an emailed statement FDA spokesperson Sandy Walsh confirmed that the FDAs decisions are all based on an assessment of the available data and whether the benefits of the drug outweigh its risks. That data can include the experience of patients like Max Rowan and Charlie. To Betty the benefits are clear even if they arent easily quantified. Rowans gait seems better. Max still doesnt need to use a wheelchair all the time bucking the prediction she heard when he was first diagnosed. However financial analysts who track the biotech sector are not optimistic about atalurens immediate prospects. Matthew Eckler a senior biotechnology analyst at RBC Capital Markets said that analysts and investors are betting the FDA will not approve the drug. The FDA hasnt said no yet. The agency still has nearly two more weeks to make a decision. Until then Betty and her sons are living with a lot of uncertainty. Im really scared that my boys will have to go off ataluren she said. Will they have access to the drug if its rejected Would their conditions deteriorate if they didnt What other options do they have Betty has no good answers. She is hoping she wont need to come up with them. DMD is a progressive disease and my boys are stable Vertin said. If we can just have a few more years with our boys or buy some time until there is a cure out there thats huge."