We’re conducting a research study about how caregiving responsibilities can affect everyday activities, routines, and participation.

If you’re a parent or legal guardian caring for a medically complex child under 18, you may be eligible to participate.

Interested in learning more? Check out the flyer below for details about eligibility and how to participate.

Your experience matters, and we’d love to hear your perspective!

https://redcap.link/4fqqz141

So my friends are planning a trip to Cali this summer and they want me to go with them. I live in NY and have Duchenne, and i've never done anything like this. Has anyone ever done something like this or have any tips on if or how this could be done? It would in involve flying cross country, and renting an RV so we can drive around the west coast for about a week. let me know if you guys know anything.

Hope this is okay to post here. I found out last year that I am a carrier for Myotubular Myopathy, and that carriers sometimes can have mild symptoms. This was validating as I have always found it hard to put on muscle, have trouble walking up stairs, and in general have weakness on one side of my body. A neurologist I met with said there's really nothing I can do except continue to exercise, and that I will not get much stronger, but it seems like there is not much research for carriers of x-linked conditions.

What I'm wondering is: are there specific kinds of PT I should look for to help with neuromuscular connection? Has anyone in this situation found certain kinds of movement to be helpful (ex. Pilates, yoga, resistance training)? I got a referral for PT but am wondering if I should look for a specialist and I'm not sure who to ask.

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https://www.fda.gov/media/195694/download?attachment=&utm_medium=email&utm_source=govdelivery

"The FDA will take the time required to build an improved framework that is scientifically sound, legally durable, operationally consistent, and calibrated to the rare disease context. It will do so transparently, with robust public engagement, and with the

sustained commitment of the agency's leadership.

The scale of the unmet need demands nothing less.

The FDA invites patients, caregivers, drug developers, clinicians, researchers, and all interested members of the public to participate in the Framework Initiative's stakeholder engagement process. Details will be available on the FDA’s Rare Disease Innovation Hub."

My 10 month old has bethlem myopathy 2 and has been on a feeding tube since birth due to dysphagia and aspiration. I’m curious if anyone else has dealt with this specifically and how long you or your little one was using an ng or g tube. I know every person is different but just curious

I had IVIG for an unrelated autoimmune condition during a hospitalization for a crisis related to said condition + obvious vulnerability with muscular dystrophy on top of it. Both my neurologist and I were curious if it will benefit my muscular dystrophy as a sort of off label sort of effect.
Wondering if anyone else has had to go through infusions of it and felt it helped with muscular dystrophy symptoms or solely just helped with the condition that was targeted.

So far, it’s barely just now kicking in for me in relieving my symptoms from my autoimmune condition. Truly wondering if it’ll have any impact on my FSHD. Maybe increased energy levels/ stable immunity will help me stretch more and move around a bit more. Who knows!

Is there any body knows hospital or physiotherapy centre or Ayurvedic centre , which treated hirayama disease..

Hey everyone, I'm A Doctor with LGMD.

Having seen firsthand how confusing it can be to navigate diagnoses, genetic reports, and the constant search for upcoming treatments, I decided to start building a dedicated mobile app to make this easier.

The goal is to create a single, easy-to-use resource that covers:

Comprehensive guides to all types and subtypes of Muscular Dystrophy

Clear breakdowns of inheritance patterns and genetics

Live updates on ongoing clinical trials and pipeline therapies

Practical resources for patients and caregivers

I’m building this as a passion project, and I want to make sure it genuinely helps rather than adds noise. If you have a minute, I’d love to know:

What do you feel is currently missing from existing websites/resources?

What’s the biggest frustration you face when looking up research or trials?

Appreciate any thoughts or suggestions you’re willing to share!

Real Patients is looking for people who have been diagnosed with a rare disease and/or their loved ones for a paid online interview $120 USD Compensation

Details

• 60-minute virtual interview

Purpose

• To understand how people living with a rare disease and their caregivers find and use health information, and their interest in listening to and engaging with a rare disease podcast

Requirements

• Diagnosed with a rare disease, or a caregiver to someone diagnosed with a rare disease
• US resident
• 18+
• English speaking

Real Patients connects people living with health conditions to research opportunities that shape how healthcare works. Founded by patients, for patients—because the best way to understand the patient experience is to talk to real patients.

Initially, I have DMD and I want to talk and maybe form a friendship with others who have DMD, of course without judgment and with understanding.

One of the reasons is that the illness is fundamental in its impact on my life, and naturally most people don't know about it. If I mention it, they won't understand, and they're often shocked. It's hard to explain the precise details and changes, not to mention that you either feel like you're pitiful or that the other person is indifferent.

That's why I often don't mention the illness, but then the matter becomes annoying because the other person assumes your situation is normal. And here's the difference, if you talk to someone who suffers from the same condition as you, your situation is understood and lived, and maybe here you feel understood to some extent.

I’m starting need more physical accommodations at work, right now it is being able to set a boundary when asked to do physical labor that puts me at risk of falling and/or restricts my ability to perform other life functions due to fatigue.

Does anyone have any experience or advice about how to navigate this process?

For context, I work for a Government conservation agency where I have to do outdoor site visits semi frequently. I don’t want to restrict my ability to do activities like walking reasonable amounts which are good for my health and day to day job, but I’m noticing I need the ability to protect myself from being asked to walk in steep terrains or lifting/carrying heavy objects…. especially when fatigued.

Hi, I was diagnosed with Duchenne as a child but a biopsy a few years later confirmed Becker. However, my disease course has mostly followed Duchenne. Anyone have a similar story?

Hiii I’m looking for an active discord server for MD. I have muscular dystrophy.

Hello!

I wanted to ask how do you approach dating and relationships?

Im 20 i have almost zero experience and im currently talking to someone and i dont really know how to approach it and how to talk about it.

I have fshd, i can walk, im not really tired, i have severe scapula winging, my arms are really weak and so are my abs, i cant run, i cant smile but i do most of things as other ppl, im just slower.

so i wanted to ask how should i approach it and when should i talk about it, i mean it is little noticable since i cant smile and i walk lowkey funny, my arms are weak and we know eachother longer and they have not said anything about me.

So i wanted to ask what are your experiences and how and when did you talked about it, im also scared of ther potencial reaction. thank u.

I have LGMD 2D, but it is quite mild.

I can mostly walk, run, hold down an office job, go out drinking and have above average muscle mass.

However, I experience daily fatigue, and sharp random muscle cramping pain in my thighs and arms daily.

I do not disclose to most people until we are really close I suffer with an MD.

I feel stuck between 2 worlds, of the disabled and able bodied life.

Mentally, because I get quite tired and am scared to be in pain randomly it puts me off starting hobbies or socialising with people because sometimes I might feel crap about 5 minutes into it.

Other times I may go out and be full of energy and have a great time.

But I don’t know when to push through or give up because my symptoms are so random and variable. I have no concrete day of pain or tiredness and so I never know what is coming that day.

I recognise my privilege in comparison to the average person who suffers with an MD, but I am annoyed that I do not have a consistent set of symptoms and thus it’s hard for me to confirm and identity a lifestyle for myself.

Finally, most people do not care / understand that you have a muscular dystrophy if you are not in a wheelchair or physically atrophied.

Even when I have explained to people I cannot help or action a certain task in work or family / coworkers question why I am not out like most young people partying and clubbing, I have to re explain I live with an MD which limits my energy.
They say they understand but just see a normal guy and ask a few months later the same things and think I’m faking.

I wonder if anyone else is on a similar boat to me, and if anything has helped them.

Has anyone done their own conversion of a van to a handicapped van? I just don't understand how these vans cost so much. Those who need them already have so many needs and expenses.

Hi everyone,

I hope you are all having a peaceful week.

I am a Product Design Engineering graduate student at the Glasgow School of Art (GSA) in the UK.

Currently, our design team is focusing on a project dedicated to children living with Muscular Dystrophy (MD). Through our academic research, we’ve learned about the incredible strength and resilience of these children, but we also realize that mainstream products and school environments often fall short of meeting their daily needs—especially when it comes to independent learning, desk-based activities, fatigue management, and everyday play.

We want to design something meaningful, practical, and truly helpful, rather than just imagining solutions from a classroom. To do this, we desperately need to learn from the real experts: the children themselves, their parents, caregivers, and teachers.

If you are a parent, family member, teacher, or adult who grew up with MD, we would be deeply grateful to hear your stories and experiences.

If you are open to sharing, we would love to know:

• The daily hurdles: What are the most exhausting or frustrating physical tasks during a normal day (e.g., getting dressed, holding objects, fatigue management)?
• School and learning challenges: How do traditional school desks, heavy writing tasks, or long classroom hours impact a child's ability to learn independently?
• Unmet needs: What products, tools, or adjustments do you wish existed to help these children play, study, and live with more autonomy and less physical strain?

You can reply directly to this thread, or if you prefer a private conversation (via Reddit DM), please feel free to reach out. Your stories will be treated with absolute respect, kept completely anonymous, and used solely for academic design research to create better solutions.

Thank you so much for reading this, and for your time and kindness. Wishing you, your families, and your little ones all the best, strength, and health.

Warm regards,

Product Design Engineering, The Glasgow School of Art

Hi, all. I’m a genetic counsellor & curious to know if you have visited a GC or are planning to. If you have visited one before, how was your experience w them? I’m curious to know how people w dmd feel about talking to GC’s and how their experience was because i hardly get real feedback other than the usual “that makes it easier to understand my report”. I want to reach out to a bigger audience w disorders and help spread awareness. Your input matters!

Hi everyone, before I start, I wanted to say that I might post a couple more questions in this subreddit if that’s okay. I was diagnosed with FSHD (Facioscapulohumeral muscular dystrophy) earlier this year, and I’m currently dealing with something that occurred recently and it’s really worrying me.

For almost a week, my entire left arm has been numb, extending into my shoulder blade and the left side of my neck. I woke up last Monday morning with the arm numb and initially unable to use it properly. I’ve had episodes like this before, but they always went away within a few hours. This time the numbness hasn’t gone away, and I feel like I’m losing some strength in the arm.

Has anyone with FSHD experienced something similar? And knowing that I have FSHD, what should I be doing or who should I be seeing about this? Should I be looking specifically for a neuromuscular specialist, neurologist, PT, or someone else?

I’m currently in Armenia, so I’m also trying to figure out what kind of specialist I should be looking for here.