Youth-Led MLD Advocacy Initiative in North Carolina

[Vrinda]

Dear Orange County Board of Commissioners, 

My name is Vrinda and I am a senior at Chapel Hill High School. I founded a youth-led nonprofit called Youth For Rare Diseases which aims to advocate for families affected by Rare Diseases which affect 1 in every 10 people. In the last month, me and 14 more local youth volunteers completed a public health initiative to raise awareness for an ultra-rare and fatal disorder known as Metachromatic Leukodystrophy (MLD). We gained about 500 handwritten signatures from North Carolina residents supporting the addition of MLD to our state’s newborn screening panel! 

We have already mailed the NCDHHS Newborn Screening Team to show our and the communities support for this cause. 

The volunteers and I care a lot about this initiative and any acknowledgment or encouragement from the Board would mean so much to us as we continue our advocacy and efforts for rare disease awareness in North Carolina. 

You can view the signature collection here if you'd like and I've also attached a PDF if needed - https://drive.google.com/file/d/1RJDuupJm2oyc7YH1m1258DeCwo5h6F7o/view?

usp=sharing

Thank you so much for your time and all that you do for our community. We really appreciate it! 

With gratitude, 

Vrinda 

984-244-9939 

2701 Homestead Rd. Chapel View Apts. Chapel Hill, NC USA 27516

fnuv...@gmail.com

[Jamezetta Bedford]

Hi Vrinda, 

Thanks for sending the pdf too (and a few more pages in another email). I am sharing your email with the Director of the Orange County Health Department. Our Board of Health may wish to review this issue. They have board members with experience re newborn screenings and rare diseases. It's impressive for high schoolers to do this type of study and advocacy! I am curious. What caused you to do this work? Was this a class assignment? Do you or the others know someone impacted by MLD? Thank you all for doing this project and continuing your advocacy. The costs of research and treatments for rare diseases are too high for commercial companies, so it is important for the federal government to provide that type of funding. Sometimes discoveries along the research for one disease open doors to other diseases too. I hope our federal government will resume increased funding core essential research In medicine, pharmacy, and many other sciences. Is there treatment for MLD if discovered early? 

Best,

Jamezetta

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From: Vrinda <fnuv...@gmail.com>
Sent: Sunday, November 30, 2025 8:02 PM
To: Jamezetta Bedford <jbed...@orangecountync.gov>
Subject: Re: Youth-Led MLD Advocacy Initiative in North Carolina

 

CAUTION: External email. Do not click links or open attachments unless verified. Report suspicious emails with the Phish Alert Button located on your Outlook menu bar on the Home tab.

Hi Chair Bedford, 

Thank you so much for your quick response. Sorry for the confusion, the PDF should be attached to this email now. Please let me know if it still did not come through and I'll be more than happy to resend it in another format if needed! 

Thank you so much for taking out time to review our initiative. It means a lot. 

With Gratitude, 

Vrinda

Chapel Hill, North Carolina

On Sun, 30 Nov 2025 at 19:53, Jamezetta Bedford <jbed...@orangecountync.gov> wrote:

I don't see the pdf. Please can you resend it? 

Jamezetta Bedford, Chair

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From: Vrinda <fnuv...@gmail.com>
Sent: Sunday, November 30, 2025 7:20 PM
To: ALL_BOCC_MANAGER_CLERK <OCB...@orangecountync.gov>
Subject: Youth-Led MLD Advocacy Initiative in North Carolina

 

CAUTION: External email. Do not click links or open attachments unless verified. Report suspicious emails with the Phish Alert Button located on your Outlook menu bar on the Home tab.

[Vrinda]

Hi Chair Bedford, 

Thank you so much for your thoughtful email and for sharing this cause with Director Stewart. I am so grateful for your support and curiosity! It would mean a lot if the Board of Health can review this effort and I hope this can become a starting point for the potential addition of MLD into the NC Newborn Screening panel.

My interest in the rare disease field began when I first interned at a rare lysosomal disorder lab at Duke Pediatrics summer of sophomore year. As I dug deeper into the research aspect of rare diseases, I also learned about the heath advocacy aspect of it as well especially, Newborn Screening. I learnt that there is a major gap in the treatment and timely diagnosis especially because many treatments work effectively only before the symptoms appear. Newborn Screening stands as a major lifeline for many future families. Ever since then, I have worked on multiple rare disease related projects from conducting research on ultra-rare disorder, NAXE deficiency to coding data collection registries for genetic counselors. 

While I do not directly know people impacted by MLD, I do realize the significance of newborn screening in lives of current and prospective parents and patients. MLD stands as in my opinion, one of the most actionable disease to be considered for newborn screening due to 3 main reasons: 

1. Implementation is already underway: New York has already implemented universal newborn screening for MLD and Illinois passed legislation for adding MLD in 2023 making it a realistic, adoptable disorder to be added to the newborn screening panel. 

2. Screening Reliability is strong: 500k babies have been tested for MLD with no false negatives or no false positives whatsoever! (reference link)

3.There is an FDA approved pre-symptomatic treatment: It has a FDA approved treatment known as Lenmeldy which only works on children before symptoms occur. The children who have been treated with this treatment have so far survived 6 years since the trial while many of the untreated children unfortunately, do not survive this long. (reference link)

And I certainly agree that the cost of treatments for rare diseases is a major concern. Even Lenmeldy, though one time treatment, stands at  $4.25 million dollars! But with newborn screening and research, we are finding out rare diseases really are not as rare as we once thought because many milder or late onset forms get missed but as more people are identified, the chances for increase in federal funding is also increasing. One of the major goals of my nonprofit is to encourage the youth to advocate for Newborn screening as well as Therapy Accessibility.

Please let me know if you need any additional information or have any other questions, I would be more than happy to provide them! Thank you so much again for your time, curiosity, and support! It genuinely means a lot! 

With Gratitude, 

Vrinda

984-244-9939

fnuv...@gmail.com