FDA Blocks Duchenne Drug for Wheelchair Patients. Texas Mom Asks: What Happened to Right to Try?

A Texas mother says the FDA cut off her wheelchair-bound son from Elevidys, raising a blunt question about what Right to Try means in practice.

Mar 25, 2026

A Texas mother is asking a question a lot of families dealing with rare diseases have been asking for years: when the clock is running out, why does the federal bureaucracy still get to move like it has all the time in the world?

Angeline Olivera of El Paso says the Food and Drug Administration has effectively cut off her 14-year-old son Ryu from Elevidys, the gene therapy developed by Sarepta Therapeutics for Duchenne muscular dystrophy. President Trump signed the Right to Try Act in 2018 to give terminally ill patients a fighting chance when the normal approval pipeline moved too slowly. Now Olivera says the spirit of that law has been buried under the same old federal caution, the same old process, and the same old answer from Washington: wait.

For families dealing with Duchenne, “wait” is not a neutral word. It means decline. It means lost muscle. It means another month you do not get back.

A Mother Making the Case Public

Olivera laid out her case in a USA Today opinion piece and in interviews with conservative outlets. Her son Ryu lives with Duchenne muscular dystrophy, a degenerative genetic disease that progressively destroys muscle function and often kills young men in early adulthood. Olivera says two of her own brothers died from the disease, so this is not theory to her. It is family history. It is grief with a calendar.

According to Olivera, Ryu now uses a wheelchair and nighttime breathing support. She says the family has already faced crushing costs tied to the disease, including a power chair costing roughly $30,000 and expensive steroid treatment. But the biggest cost is time.

“We know Duchenne is terminal. It's a devastating truth we live with every day. Muscle loss is irreversible, and delaying treatment only accelerates the decline. Every month matters. We do not have time to wait.”

That is the part the bureaucrats always seem to miss. Rare disease families do not experience delay as an inconvenience. They experience it as loss.

Why Elevidys Became the Flashpoint

Elevidys is a one-time gene therapy designed to help patients with Duchenne muscular dystrophy by delivering a gene that prompts the body to produce a shortened dystrophin protein. The FDA previously approved it for some Duchenne patients, but the agency later moved to restrict access for non-ambulatory patients after deaths tied to acute liver failure raised safety concerns.

The FDA said in a July 2025 announcement that it asked Sarepta to suspend distribution and placed trials involving related gene therapy products on hold after three deaths potentially linked to the same platform technology. The agency said the indication for non-ambulatory Duchenne patients should be restricted while it continued investigating the risk.

That is the government side of the story. Here is the other side.

Conservative reporting on the issue notes that more than 1,200 individuals had received Elevidys in trials or clinical use, while the most severe outcomes were concentrated among older and more advanced patients. Supporters of broader access argue that the risk is real, but so is the certainty of decline for boys and young men with Duchenne.

Nobody is pretending there is zero danger here. The argument is about who gets to weigh that danger.

The Core Dispute

The FDA says serious safety signals require intervention.
Families say terminal patients should be allowed to accept informed risk.
Right to Try was supposed to shift more of that choice toward patients.
Bureaucratic caution keeps pulling the decision back toward regulators.

Because of course it does.

The Senate Hearing That Put FDA Bureaucracy on Trial

The issue is not limited to one family or one drug. At a February 26 hearing of the U.S. Senate Special Committee on Aging, witnesses described an FDA process that many see as opaque, slow, and poorly fitted to rare diseases where patient populations are small and time horizons are brutal.

Townhall reported that Dr. Jeremy Schmahmann testified that ALS patients are also being denied access to a promising treatment while the agency demands yet another trial. Bradley Campbell of Amicus Therapeutics told senators that roughly 95 percent of known rare diseases still lack effective FDA-approved treatments. At the current pace, he said, developing treatments for even half of those conditions would take around 161 years.

One hundred sixty-one years.

Pretty impressive timeline, if your patient base plans to live into the 2100s.

What Families and Advocates Want

Faster pathways for terminal and rare disease treatments
Greater respect for patient choice in life-and-death cases
Regulatory standards that reflect tiny patient populations
Political pressure from HHS and Congress to force urgency

Olivera has argued that Trump was right to champion agency and choice in these cases. That is the deeper frustration here. The conservative movement fought for a principle that made moral sense: if you are facing a terminal diagnosis, Washington should not get the final word over your informed decision to try a promising treatment.

Right to Try Was Supposed to Mean Something

To be clear, Elevidys does not fit neatly into the original statutory Right to Try framework because it is not simply an unapproved investigational drug sitting in a Phase 1 pipeline. But Olivera's argument is bigger than one legal category. She is pointing to the principle President Trump put on the table in the first place.

Trump argued that families confronting terminal illness deserve a voice in life-and-death medical decisions. That was not some fringe idea. It was common sense, rooted in human dignity and the recognition that bureaucracies are built to minimize institutional risk, not to maximize hope.

And that is where this story hits a nerve on the right.

If the federal government can still tell a family facing a terminal disease that safety process matters more than timely access, then what exactly changed? If Right to Try exists in name but not in practice when it matters most, families are left with slogans while their sons lose strength.

What Happens Next

Olivera and other advocates want pressure brought to bear on the FDA and the Department of Health and Human Services. There is already bipartisan concern in Congress about how the agency handles rare disease treatments. Sen. Ron Johnson has pushed a Right to Try 2.0 concept aimed at updating the law for newer therapies and modern science.

That debate is worth having. Fast.

The federal government absolutely has a duty to warn patients about serious risk. It also has a duty not to hide behind process when families are plainly asking for the right to make an informed choice. Those are not mutually exclusive ideas. In a terminal disease context, pretending they are is how government turns caution into cruelty.

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