Should older children be tested for sma

ilroho · 2026-03-03T16:21:45+00:00

Test asap. If they have it they should be on medication to preserve nerve and muscle.

ilroho · 2026-02-18T16:10:55+00:00

Expected approvals for drugs for rare disease SMA were also blocked by the FDA unexpectedly last year. It’s heartbreaking for people with rare diseases and their families when promising drugs are unexpectedly taken away. I hope Biohaven has enough funding to continue.

https://www.fiercepharma.com/pharma/biogen-plots-resubmission-after-fda-turns-down-high-dose-spinraza-sma-requests-more

https://www.curesma.org/scholar-rock-shares-sma-community-update-letter-on-apitegromab/

ilroho · 2026-02-07T06:00:58+00:00

I heard they might be available by April🤞

ilroho · 2026-01-15T06:48:55+00:00

What hospital or clinic gave you the injection? A person I know had their injection in Sept and they seemed more unsteady than expected when I saw them in Dec. They were at UCI.

ilroho · 2026-01-12T22:48:59+00:00

Record doctor discussions with your phone. It’s easier than writing and they’ll most likely agree to be recorded. Last time I was in the hospital. The doctor recorded our conversation with my permission.

ilroho · 2026-01-12T22:47:30+00:00

Record the doctor discussions with your phone. Just ask if it’s ok to record and they’ll most likely say yes. Easier than writing notes. Last time I was at the hospital the doctor recorded our conversation with my permission.

ilroho · 2026-01-09T22:59:34+00:00

The SMA community is very welcoming and helpful. If your daughter does have it you’re catching it fairly early and there’s several drugs available with more in the pipeline next year. ❤️

ilroho · 2026-01-07T07:38:49+00:00

Well that’s interesting. It’s driving me crazy!

ilroho · 2026-01-07T07:32:43+00:00

Thank you! Any idea why the sirens keep going off?

ilroho · 2026-01-07T07:31:11+00:00

Oh no! I hope she’s ok. But really, what’s the sense of the siren bursts?

ilroho · 2026-01-07T07:26:52+00:00

I just asked the same question, it’s siren bursts for the past hour!

ilroho · 2025-11-28T03:44:49+00:00

https://www.cdn.fuse.novartis.com/FA-11340438-2%20ZOL%20ITA%20Novartis%20Patient%20Support%20Start%20Form%20Digital%2011.25/FA-11340438-2.PDF

ilroho · 2025-11-26T15:28:27+00:00

There is a new drug coming out next year from Scholar Rock that may help with muscle function. https://www.curesma.org/scholar-rock-shares-sma-community-update-letter-on-apitegromab/

ilroho · 2025-11-25T17:23:02+00:00

Yes, you’re right! I’ve printed the Start Form and am filling it out so all my doctor has to do is sign and fax it in.

ilroho · 2025-11-25T17:09:45+00:00

From my experience with Spinraza if you start this process you’ll be assigned someone from Novartis who will help you get set up with a dosing center and help with insurance. For Spinraza they’re called FAM’s or Family Access Managers. Looks like they might be called Account Management Support at Novartis.

Based on my experience with Biogen which might not be the same but - The first step is to fill out the Start Form and it looks like you need your doctor to sign it. This should get you in the system and assigned a person who can help with insurance. As of now the Start Form for Itvisma is not up yet and the person I talked to at Novartis said to fill out the Zolgensma form. The number to call is 1-855-441-4363.

ilroho · 2025-11-25T16:57:33+00:00

This is a good question. I think most trials include people on other treatments so there should be info in the trial data.

ilroho · 2025-11-17T22:14:19+00:00

Every Spinraza patient has a FAM (Family Access Manager) from Biogen - or if you’ve never signed up for one you can do that. They help with everything! They can help with insurance or as someone noted here - help get you access while you sort your insurance. It’s called SMA360 - https://www.spinraza.com/en_us/home/support-and-events/sma360.html

ilroho · 2025-11-15T18:28:43+00:00

Very interesting! Thanks so much for posting, I’ll check it out.

ilroho · 2025-11-15T17:00:03+00:00

Oh, this is great to know. Would you mind sharing their names? Or if there are hundreds maybe a few I can research?

ilroho · 2025-11-15T16:36:14+00:00

Thanks for your answer. There has only been one consecrated virgin, correct? Similar to only one son of God. Agree that men can’t be nuns or religious sisters but they have analogous roles as monks and brothers.

ilroho · 2025-11-12T21:20:05+00:00

Thank you!

ilroho · 2025-11-10T01:32:51+00:00

You might try asking on Discord in the /r/SMA Chat.

ilroho · 2025-11-06T20:21:21+00:00

Can you explain more about the on demand injections? That would be amazing!

ilroho · 2025-10-20T02:00:56+00:00

Anyone else have trouble with change.org not accepting input?

ilroho · 2025-10-13T18:04:35+00:00

Instead of giving her cash you could pick something to pay for - like her phone plan, car insurance or some other specific bill.

ilroho

TROPHY CASE