by Linda Childers, January 26, 2016 | From: NBC NEWS

When Damon Woods of San Diego, Calif, was diagnosed four years ago with Duchenne muscular dystrophy, a rare and fatal muscle disorder, his mother, Charaine vowed to do whatever it took to save his life.

 This week, Woods and other families were dealt a blow when the Food and Drug Administration (FDA) declined to approve Kyndrisa, generically known as drisapersen, an experimental drug that had shown promise in treating Woods and other boys affected by Duchenne muscular dystrophy.

 According to the national non-profit, CureDuchenne, Duchenne is the most common and lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 patients worldwide, primarily boys and young men, and there is currently no FDA-approved therapy specifically designed to treat Duchenne.

 A New Medication Provides Hope

 After her son, Damon, was diagnosed with Duchenne muscular dystrophy at the age of nine, Charaine watched helplessly as her once vibrant child became weaker and began falling. She was only too aware of how most boys diagnosed with the condition suffer from muscle degeneration that ultimately affects their heart and breathing muscles, leaving them unable to walk or breathe.

• Damon and Charaine Woods with Sen. Joel Anderson’s Legislative Director Craig Wilson

Then in 2013, Woods learned about a new clinical trial for drisapersen (Kyndrisa), a high-tech drug designed to fix the underlying genetic defect causing the progressive muscular decline seen in children with Duchenne. Manufactured by BioMarin Pharmaceutical in Novato, Calif., the drug underwent clinical trials at the University of California, Davis, and a handful of other research centers across the country. Woods made the 500-mile trek with Damon to Sacramento each week, believing it was worth it if the medication could give Damon a shot at a long and productive life.

 “After a month of being on drisapersen, Damon was able to walk upstairs and he wasn’t falling anymore,” Woods says. “He was able to be independent and the drug seemed to be slowing the progression of the disease.”

 “We are physically and emotionally exhausted as we not only fight for a treatment but as Damon battles every day of his life with this disease.”

Dr. Craig McDonald, professor and chair of the Department of Physical Medicine Rehabilitation at UC Davis, was quoted in a news release as saying that drisapersen was “the most exciting treatment approach I have witnessed in my career for Duchenne muscular dystrophy,” and that he was hopeful “it will delay many of the disease’s manifestations and ultimately improve life expectancy for patients.”

 Woods was optimistic drisapersen would receive approval from the Food and Drug Administration (FDA). In November, she spoke at the FDA advisory committee’s hearing regarding the investigational treatment helping her son, and there wasn’t a dry eye was in the house.

 Rallying After a Setback

Hope turned to disappointment this past week when the FDA issued a statement saying they “concluded that the standard of substantial evidence of effectiveness has not been met” and that the “treatment is not ready for approval in its current form.”

“I am very disappointed about the FDA drisapersen decision,” says Debra Miller, co-founder of the Newport Beach, Calif., non-profit, CureDuchenne. “As a parent and patient advocate, I believe there was ample evidence to approve this drug on a conditional basis while they confirm the effectiveness and safety over a longer term study.”

Damon with the Californian Flag

Woods also expressed sadness at the news.

“We have done everything in our power to beg for a treatment for Damon’s life and sadly we have been rejected,” Woods says. “We are physically and emotionally exhausted as we not only fight for a treatment but as Damon battles every day of his life with this disease.”

BioMarin said Thursday they will continue clinical trials of drisapersen and “will work with the FDA to determine the appropriate next steps regarding this application.”

While Damon is still receiving drisapersen from BioMarin as they collect more data, Woods is uncertain what the future holds for her son and other boys affected with Duchenne muscular dystrophy.

“We have seen the difference this medication makes in Damon’s life. I know that without drisapersen he will become a boy trapped in his own body with absolutely no control over it, completely paralyzed most likely by the age of 17 or 18,” Woods says. “I plan on doing everything I can to help get drisapersen approved. It’s not just for Damon, but all the children who are waiting on a treatment for this life-stealing, devastating disease.”

More Dying Patients Being Denied Access to Dr. Burzynski’s Life-Saving Treatment 518 BY ANH-USA ON JULY 7, 2015 STOP CRONY HEALTHCARE

The FDA is effectively signing the death warrant of more patients by denying them access to Dr. Burzynski’s antineoplaston cancer treatment—for no rational reason whatsoever. Please help. Action Alert!

Over the years, we’ve covered the FDA’s attacks on Dr. Stanislaw Burzynski, the trailblazing cancer doctor best known for his discovery and development of antineoplastons (ANP), which are peptides and amino acid derivatives that activate tumor-suppressing genes. Independent research has confirmed antineoplastons to be an effective cancer treatment.

As we’ve reported in the past, the conventional medical establishment has, through a succession of relentless attacks, slowly choked off patient access to antineoplastons. In July 2012, after years of failed legal attempts to shutter the Burzynski clinic completely, the FDA told Dr. Burzynski he could no longer accept children for treatment with antineoplastons during the current FDA trial. In January 2013, this ban was extended to adults. This means that under current FDA restrictions, no new patients can be treated with antineoplastons. Terminal cases who could be saved will instead die.

Since the FDA has formally forbidden new patient access to antineoplastons, the only hope of dying patients is to convince the FDA to grant a “compassionate use” or “single-patient protocol” exemption via its expanded access rule. This rule allows for the case-by-case use of an experimental or unapproved drug outside of a clinical trial if a patient has a serious or immediately life-threatening disease or condition, and has no other treatment options left.

For an individual patient to be granted access to an experimental drug in the expanded access rule, a doctor need only conclude that the experimental drug does not pose a greater risk than the disease itself. Despite this rule, the FDA has, on a number of occasions, refused to grant access to antineoplastons for patients for whom the treatment is the last shot at living. In one tragic case, a five-year-old diagnosed with aggressive brain cancer died while waiting for the FDA to approve ANP treatment. The FDA eventually did grant the child compassionate use, but by that time he was already brain dead.

In May of this year, a patient with glioblastoma (GBM) was denied access to Dr. Burzynski’s treatment because the FDA judged that “the potential benefits [did not]justify the potential risks of the treatment.” GBM is a highly malignant form of brain cancer and extremely difficult to treat. Apparently death is less risky for these patients than a natural and effective treatment which has a long history of safe use.

What is both confusing and astonishing is that the FDA representatives who denied the request did not challenge the fact that the antineoplaston treatment has no significant additional side effects when compared to standard cancer therapies. The FDA’s main point of contention seems to be that they consider antineoplastons to be an ineffective treatment for GBM, hence they see no perceived benefit to the treatment. The actual effectiveness of the treatment is not in doubt, however, having been used for many years, but that is supposed to be determined by the current FDA-approved trial.

The government actually agreed to the trial—but only if antineoplastons were combined with conventional chemotherapy, just to muddy the results and protect chemo’s primacy as a cancer treatment. And given the government’s attitude, one can only wonder if the results of the trial will ever see the light of day.

Why then is the FDA denying patients access to a potentially lifesaving treatment when nothing else works? These are exactly the kinds of situations that compassionate use and expanded access were made for—patients with no other options who want access to experimental treatments. But rather than give patients one last chance, the FDA effectively signs their death warrant by denying access to Dr. Burzynski’s antineoplaston treatment. This has to stop.

A public outcry against this madness can make a real difference and save lives. Last March, after a similar ANH-USA grassroots campaign to grant dying patients access to Dr. Burzynski’s treatment, the FDA quietly granted eight patients compassionate use exemptions.