Cure MLD

We are on a mission to #cureMLD, a #lysosomalstoragedisorder (LSD) & #leukodystrophy impacting kids & adults. #genetherapy #advocacy

[Cure MLD @cure_mld]

Calling all MLD families! Cure MLD’s community survey is moving to a new home. We are excited to announce the launch of MLDCures, a survey platform we created with @RareDiseases and @caljoyfndtion.

Fill out your information in our new survey home HERE: https://t.co/GjCzR4oqUs

[Cure MLD @cure_mld]

Recent article alert! The importance of early diagnosis and views on NBS in MLD: results of Caregiver Survey in the UK and Republic of Ireland! Read more at https://t.co/kx0qjtARdV

[Cure MLD @cure_mld]

In the News! An international study of caregiver-related burden and quality of life in MLD. Researchers found that MLD can negatively impact a caregivers health, relationships, and professional status. You can read more about caregiver burdens with MLD at https://t.co/Q3DpSFvGD9

[Cure MLD @cure_mld]

Today is #GivingTuesday. A donation toward Cure MLD will help us best support families, provide resources to those in need, and advocate toward treatments for MLD and NBS. If you choose to give on this Day of Giving, you can donate at https://t.co/3hF7KylfXI

[Cure MLD @cure_mld]

HAPPENING TODAY at 11am EST! Tune into the EL-PFDD scientific follow-up led by clinical experts discussing therapies, drug development, and clinical trial opportunities. Check out the Scientific Workshop at https://t.co/Jlq8UnsGVh

#MLD #leukodystrophy

[Cure MLD @cure_mld]

This Friday from 11am-1pm the #MLD community is hosting an EL-PFDD follow-up with clinical experts! Register and view the MLD Scientific Workshop at: https://t.co/tUwNYP0zmQ

[Cure MLD @cure_mld]

HAPPENING FRIDAY!

The MLD community is hosting a follow-up to the EL-PFDD with clinical experts to discuss insights from the meeting on Friday, November 18th, from 11am-1pm EST.

Register and view the MLD Scientific Meeting at:
https://t.co/Jlq8UnKijR

[LDNBSActionNetwork @LDNBSAction]

HAPPENING NOW: Facebook Live - Meet Newborn Screening Advisory Board Members

https://t.co/Pe5HHdZTPT

Find out what state NBS Advisory Boards do and how you might be able to get involved today!

#NBS #newbornscreening

[Cure MLD @cure_mld]

Join us this Wednesday at the LDNBS Action Network's Facebook page to learn more about newborn screening advisory boards and how YOU might get involved!

[LDNBSActionNetwork @LDNBSAction]

Join @LDNBSAction for a Facebook Live on October 26th to learn from #NewbornScreening advisory board members!

Come listen, ask questions, and be better prepared to advocate for NBS conditions to be added in your state!

More info here: https://t.co/WlpXY27PXB

[Cure MLD @cure_mld]

HAPPENING TODAY!

The MLD EL-PFDD is happening LIVE!

Go to https://t.co/ajljudqLgV to 1) view the meeting TODAY, October 21st, and/or 2) participate directly by calling or writing in LIVE. We want to hear what matters most to you about disease burden and hopes for treatment.

[Cure MLD @cure_mld]

Have you registered for the Oct. 21st MLD EL-PFDD yet?

EL-PFDD meetings give the FDA, researchers, biopharma companies, health care providers, and federal regulatory partners an opportunity to hear directly from patient families.

Pre-register here: https://t.co/jWHJiFN4dn

[Cure MLD @cure_mld]

Cure MLD is proud to partner with @UlfUnited on #LeukodystrophyAwarenessMonth efforts! We worked with ULF & partner orgs to create a video about pronouncing the word "leukodystrophy." #SayItRight

Watch the video here: https://t.co/o9DcTQHaOQ

[Cure MLD @cure_mld]

💙

[Orchard Therapeutics - U.S. @orchard_tx]

Thank you to @cure_mld and @LDNBSAction for getting the Longfellow Bridge in Boston lit up in blue earlier this month, just days after @MassGovernor issued a proclamation recognizing September 2022 as #LeukodystrophyAwarenessMonth. Read more: https://t.co/kDr5V1BSky

[Cure MLD @cure_mld]

Did you know? September is also Newborn Screening Awareness Month!

[ULF @UlfUnited]

Leukodytrophy Awareness Month Fact of the day!
Visit https://t.co/nLRD9Hvg8w for ideas you can use to raise awareness.

#ULF #LeukodystrophyAwarenessMonth #UnitedLeukodystrophyFoundation #notgivingup #ShineALightOnLeukodystrophy

[Cure MLD @cure_mld]

Read all about it! https://t.co/pwoHcoCJRr

"Since the state’s new screening policies went into effect, four babies with Krabbe have been diagnosed. “It’s stunning, for a rare disease that has a 1 in 100,000 incidence rate,” Brackbill said."

#newbornscreening #Leukodystrophy

[Cure MLD @cure_mld]

#ICYMI Last week, the MLD community hosted a webinar about how to participate in the upcoming EL-PFDD ! You can view the webinar recording here: https://t.co/3tMWy6tt2E

We HIGHLY encourage participation! This meeting is for you to teach decision-makers about how MLD impacts you.

[Cure MLD @cure_mld]

@aidanslaw Congratulations!

[Genetix Biotherapeutics @genetix_biotx]

#ICYMI: We're delighted @US_FDA has approved our #genetherapy for the treatment of early, active cerebral #adrenoleukodystrophy (CALD) in boys 4-17 years of age. https://t.co/vI9uHViULt

[ULF @UlfUnited]

Join the #SayItRight campaign! There are over 50 types of leukodystrophy and we want to show the world how to #SayItRight. Please take a moment to record a video saying your specific type to potentially be included in our #SayItRight video. Submit by 9/26. https://t.co/CWDTbKLOiJ

[Cure MLD @cure_mld]

INCREDIBLE NEWS for #Leukodystrophy #GeneTherapy:
bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD)

Congrats, @bluebirdbio, on this historic achievement.

Learn more: https://t.co/DtJddmrmHy

#ALD

[Cure MLD @cure_mld]

The Longfellow Bridge in Boston, MA, lit up in blue last night for #LeukodystrophyAwarenessMonth (thanks @MassDOT​!) And @MassGovernor Charlie Baker issued a proclamation recognizing Sept. 2022 as Leukodystrophy Awareness Month!

Read more: https://t.co/4kHJgGQ4Ty

#ShineALight