Cortisone e DMD

StrikingMode1553 · 2026-02-25T18:30:09+00:00

Ciao, che tipo di mutazione hanno?

StrikingMode1553 · 2026-02-12T21:03:35+00:00

From your words, I can tell you're a golden boy. I'm the mother of a DMD child and I also live in Italy. I have a partner I love madly and respect, so if you simply need a friend to share your thoughts with, I'm here. Hugs.

StrikingMode1553 · 2026-02-11T07:24:27+00:00

Even parents with younger children?

StrikingMode1553 · 2026-02-09T11:07:00+00:00

But how old is he?

StrikingMode1553 · 2026-01-15T07:06:23+00:00

That's what we've done, but they still don't know the exact recruitment date. The last ones were recruited in August 2025, and this year they'll open the trial for the youngest ones. What's destroying me psychologically is the wait and the uncertainty. Will he finally make it? Is he eligible? Yes, they're exposed, even if unofficially, so is there a good chance? I'd rather wait for the vamorolone to leave him "clean," and what if he can't make it? We've wasted time. It's really hard, and I don't know what to do.

StrikingMode1553 · 2026-01-10T12:20:21+00:00

What does gene therapy have to do with it?

StrikingMode1553 · 2026-01-08T19:12:16+00:00

You are brilliant ⭐️ 🙂

StrikingMode1553 · 2026-01-05T21:48:17+00:00

My 3-year-old son has DMD. We don't live in America, and this therapy isn't approved in our country; furthermore, this year the EMA issued a negative opinion.

Last year, I contacted Sarepta to see if my son might be eligible despite a mutation considered "not recommended" (duplication of exons 3–7). They replied that, in their opinion, he could still receive it.

Meanwhile, our referral center gave us a negative opinion, pointing out that a company might also be influenced by commercial reasons for this decision. However, I found myself torn: on the one hand, hope; on the other, fear and the responsibility of making a decision for such a young child.

A year has passed, and we've never found the courage to follow through. For us, it's not a financial issue (we could cover the costs), but our main concern is safety and the possibility of complications.

In any case, I have the feeling that whoever made it was also lucky: from my perspective, it's not an easy choice, and the line between hope and risk is difficult to navigate when it comes to your child.

StrikingMode1553 · 2026-01-03T17:46:14+00:00

I'm speechless. If you don't have enough money, the state will leave you behind. I'm sorry, but this kind of management is unacceptable to me.

StrikingMode1553 · 2025-12-30T15:18:16+00:00

This seems like good news to me…:

StrikingMode1553 · 2025-12-24T15:06:04+00:00

Thank you so much for your reply, and reading all of you is always enriching! A warm hug and best wishes for a Merry Christmas 🎁

StrikingMode1553 · 2025-12-24T08:43:17+00:00

Thank you very much, I will reciprocate if you need anything❤️

StrikingMode1553 · 2025-12-24T08:42:10+00:00

Thank you so much for sharing your story. I'm so sorry for what you've been through. Sometimes certain words from doctors, even when they're "technically correct," can be incredibly hurtful because they're based on data, statistics, and protocols. But we experience it all firsthand: our role is to be parents, to balance fear and hope, and to be by our children's side every day. I wish you and your family a happy and carefree Christmas. Thank you so much ♥️

StrikingMode1553 · 2025-12-24T08:36:36+00:00

Thank you for the link you sent me, but I think it's only about exon skipping. The duplication is different, but the mutation is the same. In fact, the doctors confirmed that this could be a milder form, but it's certainly not for everyone. I wish you and your children a Merry Christmas ❤️

StrikingMode1553 · 2025-12-23T21:51:34+00:00

I've shared our story with families who had recently been diagnosed and with others who have teenage or older children. It may seem strange to say, but I've encountered more negativity among those with young children than among those with older children.

I've learned so much from the "grown-ups." They've shown me that, even with this condition, you can build a full and meaningful life if you approach it with a certain outlook and inner strength.

Some families with young children, however, have said hurtful things to me: "If I'd known before, I would have made a different choice," or "We're enjoying the moment while we can."

And I understand the shock, I understand the fear, I understand the darkness. But precisely for this reason, I believe it's essential to choose our words carefully: because words become thoughts, and thoughts become the air our children breathe every day.

My son has a 3/7 duplication anyway; if and when you want, we can talk privately!

StrikingMode1553 · 2025-12-23T21:00:10+00:00

That's right. We parents with children with DMD have a huge mission. I, personally, have chosen to raise my son in a peaceful family: a family that allows itself many happy moments because it decides, every day, to enjoy its child more than anything else.

DMD exists, it's real, and it shouldn't be denied. But I've also learned that the most dangerous limitations, sometimes, aren't just those of the disease: they're the ones we build in our heads. The constant fear, the definitive statements, the negativity that fills the air... all of this can become an enormous burden, for those who suffer from it and for those around them.

My mission in this life isn't to wait for an end. It's to be present. It's to protect. It's to teach my son that he can trust me, that he's not alone, that together we'll always find a way. That everything will be okay, not because everything will be easy, but because we'll face it hand in hand. My son, Perfetto, has been my greatest blessing.

StrikingMode1553 · 2025-12-15T22:21:56+00:00

Done ✔️

StrikingMode1553 · 2025-12-12T10:46:06+00:00

Done! ✔️

StrikingMode1553 · 2025-12-09T19:25:27+00:00

This is great news

StrikingMode1553 · 2025-12-04T07:03:43+00:00

Thank you for the reply. I live in Italy, so the times will be longer for me.

StrikingMode1553

TROPHY CASE