Friday was one of those days, meetings in Washington all week and anxious to get home. Traffic was a mess, Friday evening flights out of DC chaotic. I worried I would miss my flight, the last one to Cincinnati. I attempted to guesstimate the amount of time to get through the security line, grab a salad, and still make the flight. I made it just in time to learn my flight was delayed.

Plan B. I made my way to the small tables, unwrapped my salad, and watched as the board changed. Most of the flights going out of Gate 35X were delayed, one after another, gate personnel giving rather lame excuses about weather, wind, maintenance…or maybe just Friday night in DC and big jets take precedence over commuter jets to small cities.

Tables were hard to come by and mine had an extra chair. Another woman traveler, with a delayed flight to Kansas, asked if she could sit. We lamented our delayed flights and compared notes about travel. We did the typical ‘why are you in DC’ sort of conversation and it turned out this woman was a commuter – DC during the week and Kansas City on weekends – her career focused on insurance and reimbursement. We had a good, long talk about durable medical equipment and the apparent lack of knowledge, insight, and understanding as families are frequently denied essential equipment.

It was refreshing to speak with her. She knew about Duchenne (and did not confuse it with MS!) and knew several families. She said there was ‘lots of hullabaloo’ in Duchenne these days. I smiled and said, “Hullabaloo is a good word actually.”

It means commotion, noise, outcry. After over 20 years in the Duchenne space, it means people are finally starting to hear our pleas, hear our stories, and slowly understand Duchenne.

What’s all the hullabaloo about?

We have 27 companies working on interventions.

Two Advisory Committee meetings are anticipated in November.

Both companies have drugs that target the same mechanism of action (exon skipping) and same molecular target (exon 51).

In other rare diseases, there are companies competing (enzyme replacement for example), but they have not conducted trials at the same time, had Advisory Committee meetings scheduled back to back, both seeking approvals and competing for the same patients.

The need for options.

From the side of the patients, all young men and women with Duchenne need and deserve options. While we can sit around all day and discuss backbone chemistry, dose, benefit, and side effects, it all comes down to a family meeting with their clinician, discussing options.

  • What is best for me, for my son (or daughter)?
  • What are my options?
  • How does it work? Will it affect all muscle cells? Degenerating fibers? What about the heart?
  • What did you learn from the clinical trials?
  • How long will it take before I (we) may see an effect?
  • What is the most effective dose?
  • What should I be aware of in terms of side effects?
  • If side effects, are they manageable?
  • What makes most sense for my child (for me?) today, now?
  • If this does not work, is there another option to consider?

We do this every day, with so many things we face in life. Lay out possibilities and explore options. Weigh the benefits and risks. Navigate insurance options. Figure out what will be required, what is needed, and how we will fit this into the context of our life, our family, and our jobs.

More hullabaloo.

Exciting times. Complex times.

Duchenne has come of age and the complexities of our world are wonderful, scary, and most certainly accompanied by some degree of apprehension. We have been asking this one single question since we gathered at PPMD’s first conference in Florida in 1994: WHEN?

For this intervention, skipping exon 51, the answer is not the hard to define ‘soon,’ but NOW!

We are a community united behind treatments for ALL. Since our formation in 1994, PPMD has sought treatments and insisted on optimal care for ALL people with Duchenne. It is why we are working so diligently with both BioMarin and Sarepta in preparation for their possible Advisory Committee meetings later this fall.

We believe that all safe and effective treatments deserve quick approval. We believe that patients, that our loved ones living with Duchenne, deserve options. Maybe you like Tylenol over Advil – you have a choice to take the drug that best treats your symptoms. We need to support these companies as they approach the finish line so that the decision on what treatment you or your child is prescribed is a decision you and your physicians make. Not a situation where you have no options.

Not only is PPMD working with industry to get their treatments to market, we’re looking at what’s next in terms of access, reimbursement, and labeling. This is a marathon, not a sprint and PPMD is in it to win it. We believe that the path these first approvals forge is a path for other potential treatments to follow.

Of course there’s noise, commotion, hullabaloo. Because it is the dawn of a new era…Treatments.

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