Center for Duchenne Muscular Dystrophy at UCLA

The World Duchenne Awareness Day 2024 theme is ‘Raise your voice for Duchenne’. World Duchenne Awareness Day (WDAD) is an annual event held on September 7. With this year’s theme, WDAD supports creating a society that provides equal opportunities for all.
This year’s theme emphasizes the importance of amplifying voices to advocate for the rights, inclusion and well-being of people living with Duchenne muscular dystrophy (DMD) and other dystrophinopathies.
On September 7 we invite everyone, irrespective of their personal connection to Duchenne, to join in creating a more inclusive world where people living with disabilities are empowered to thrive.

Thursday, September 5, 2024, at 1:00 PM ET.
There are so many ways you can raise your voice for Duchenne—register today and learn how you can get involved with PPMD’s advocacy efforts:

Webinar: PPMD Advocacy - Raising Our Voices in 2024 and Beyond As we approach World Duchenne Awareness Day, join PPMD for a special webinar, “PPMD Advocacy: Raising Our Voices in 2024 and Beyond,” on Thursday, September 5, 2024, at 1:00pm ET. PPMD Advocates from throughout the...

Register here: https://www.jettfoundation.org/event/community-webinar-debunking-tracheostomy-myths?fbclid=IwY2xjawE5zJNleHRuA2FlbQIxMAABHT3ZsqusSi0_OWSM8Y9T0LVGSy5DPnosx0fKY6X8R9JCtzG9C2zil_jypg_aem_ScbHJ5lt5Vl_e88_655Pdg

This Thursday, August 29th, 2024 1:00 - 2:00pm PT

Gene Therapy Series Part 1: New Treatments in DMD: Which One is Right for Me? | Muscular Dystrophy Association Attendees will have the opportunity to ask questions during the live Q&A. There is no cost to members of the MDA community or healthcare providers to attend the event, however registration is required. Not able to attend on August 29? This webinar will be recorded for on-demand viewing. Please regis...

Rest up from all that Olympic cheers because the Paralympics are right around the corner! Opening Ceremony: August 28, 2024 11:00am PDT (8pm local)
Check below for some key dates:
https://links.pages.support.ucla.edu/els/v2/PG8YCg~yPvtb/NFh6eUVxOVdDUkV6NTNFWGRxQXpjblVLSW9sUG91L1ZjUUYxN2JpYXBFWTVock1yREZWY25SUjIvSzFhK2ZLeTdWb2FUbmlWNFZvR0hBcTJsWW9UV09PRndIY0VpNGg2ejZ4d1hVbGlLaUE9S0/

Newborn screening for Duchenne added in Massachussets. Working on California now!
Full press release here: https://www.mass.gov/news/governor-healey-signs-maternal-health-bill-expanding-access-to-midwifery-birth-centers-and-doulas-in-massachusetts

The World Duchenne Awareness Day 2024 theme is ‘Raise your voice for Duchenne’. World Duchenne Awareness Day (WDAD) is an annual event held on September 7. With this year’s theme, WDAD supports creating a society that provides equal opportunities for all.

This year’s theme emphasizes the importance of amplifying voices to advocate for the rights, inclusion and well-being of people living with Duchenne muscular dystrophy (DMD) and other dystrophinopathies.

On September 7 we invite everyone, irrespective of their personal connection to Duchenne, to join in creating a more inclusive world where people living with disabilities are empowered to thrive.

Becker Education and Engagement day will take place on September 28th. Register here: https://beckereducationandengagement.com/registration/

Patients dosed in Phase 1/2 INSPIRE DUCHENNE trial of SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne); Dosing was well tolerated in all patients with initial data expected Q4 2024. Solid plans to expand patient dosing with additional clinical trial sites in the U.S., Canada and Europe and plans accelerated production of multiple GMP batches of SGT-003 to support trial expansion. Full press release here:

Solid Biosciences Reports Second Quarter 2024 Financial Results and Provides Business Updates | Solid Biosciences Inc. The Investor Relations website contains information about Solid Biosciences Inc.'s business for stockholders, potential investors, and financial analysts.

https://aviditybiosciences.investorroom.com/2024-08-09-Avidity-Biosciences-Announces-Positive-AOC-1044-Data-Demonstrated-Significant-Increase-of-25-in-Dystrophin-Production-and-Reduction-of-Creatine-Kinase-Levels-to-Near-Normal-in-People-Living-with-Duchenne-Muscular-Dystrophy-Amenable-to-Exon-44-S

Avidity Biosciences Announces Positive AOC 1044 Data Demonstrated Significant Increase of 25% in Dystrophin Production and Reduction of Creatine Kinase Levels to Near Normal in People Living with Duchenne Muscular Dystrophy Amenable to Exon 44... Delpacibart zotadirsen (AOC 1044) delivered unsurpassed muscle concentrations of PMO of 200 nM after three doses of 5 mg/kg Statistically significant 37% increase in exon 44 skipping and up to 66%...

Join CureDuchenne on Friday, August 16th, 2024, at 9:00 AM PT | 12:00 PM ET for an exclusive webinar with Avidity Biosciences.
Register here: https://l.facebook.com/l.php?u=https%3A%2F%2Fus02web.zoom.us%2Fwebinar%2Fregister%2FWN_fxeRaHKzRdGUMB7YMRrPhg%3Ffbclid%3DIwZXh0bgNhZW0CMTAAAR2nTXeycKbNrBMIAmd26kZP5fw5tu36qGprdr9pfYAWbDToaApnLBVq7rQ_aem_F7LIV00s2M-JfOseVqTl1w%23%2Fregistration&h=AT1CdOwxnbAYfuxocrh3LEQGHB6zkxk9kuhQUrBBpPNrndZwQyiINmMr0Z4SVsbZiG2ANNHPyCqAS8tI9-SusaG6byExxeHWCUVhpNtJiPYFVYwnXqnv_6MBxZb1Vlr1iLp0qO1WYw&__tn__=-UK-R&c[0]=AT3ST5vtUQ1E9EHF298YB9RVzxwkZ7JcWv8Txa2qHJD2gLoFH1U_ebcJ-_ymMhyEoBgq_F0Nk6hpOrQkEFmdEw_p3KHF2t9PwzUUuxVSNk0H8jbV1HrMDEHnSRG-S1MvByaWyKFm7Oo3uJG1KQBP-X1yQK6e-k38wKngHXda4W4NeFMQAFfZTEohGs7jLA-xrdJbykgyYSR3Upwq0aSh_Xo279gp0n_sbfCv

https://www.parentprojectmd.org/ppmd-urges-fda-to-conduct-a-full-review-of-ataluren-translarna-following-resubmission-of-nda/?fbclid=IwY2xjawEopONleHRuA2FlbQIxMQABHbR60krDSRYfi0uTTlD3VoVQpVp_1o6BBygkE8la2p2Ys2AMLEhpVcbdsQ_aem_02eD_mX9Ag9AOHkRweTURg

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon...

PPMD and Edgewise Therapeutics will host a webinar on August 7th, 2024, at 1 PM ET to learn more about Edgewise’s ongoing clinical trials, which aim to protect muscle in individuals living with Becker. Register here: https://parentprojectmd-org.zoom.us/webinar/register/WN_05n0O_DXQQ26mht3v1pgNQ?fbclid=IwY2xjawEeQKtleHRuA2FlbQIxMAABHYjzz7eSvbzvHp4Hpb-riVI20oh6kcJHc6HmvJ8kt5zBFo7f4OQ_UCKKiA_aem_RpvKerwIbmwdnJ3J1S6SNg #/registration

The Rare Artist program, powered by the EveryLife Foundation for Rare Diseases, was established in 2010 to showcase the talents of those impacted by rare diseases. The annual contest provides a national platform for artists to advocate through visual artwork and poetry. Learn more here: https://rareartist.awardsplatform.com/?utm_campaign=2023%20Rare%20Artist%20Contest&utm_medium=email&_hsenc=p2ANqtz-_Z7_w35yAnunhv7RyhSmX1UR7KXNM88ombuYdRoOdvyPfbcEl6Wz80j9PI4CxBrEYEjewXbbvL6QLCg3nflIy_KndAgg&_hsmi=318309042&utm_content=318309042&utm_source=hs_email

hs-22731230.f.hubspotemail.net

In a letter to the community yesterday, Pfizer
shared that they have made the difficult decision to discontinue their Gene Therapy program for Duchenne.

Becker education and engagement day September 28th. Register here:

Becker Education & Engagement BEED began in 2023 when a group of individuals living with Becker, advocacy groups, clinicians, and researchers in Becker came together to design an opportunity for those in the Becker community to connect. The event was designed to foster a sense of community for people living with Becker and their...

Duvyzat is now available commercially in the US. This is the first nonsteroidal treatment approved for patients six years of age and older with DMD regardless of genetic mutation. Read ITF press release here for more info and support available. Talk to your Neuromuscular care team if you are interested in starting this therapy.
https://itftherapeutics.com/documents/ITF-Therapeutics-LLC-Announces-US-Commercial-Availability-of-DUVYZAT.pdf?fbclid=IwY2xjawEPjCZleHRuA2FlbQIxMQABHYspXsZUtAFUO8QGQo_jTrD626U0AhDdzDUfjteogndOEVGq-FpWFGBRfg_aem_RSaPt836j3Rk-3B63F2aLw

itftherapeutics.com

https://www.fda.gov/news-events/fda-voices/fda-rare-disease-innovation-hub-enhance-and-advance-outcomes-patients?utm_medium=email&utm_source=govdelivery

FDA Rare Disease Innovation Hub to Advance Outcomes for Patients The FDA plans to establish a Rare Disease Innovation Hub. Our vision and goal for the Hub is ultimately to improve outcomes for patients.

Duchenne and School Challenges Duchenne and School Challenges I wish they understood what it’s like to be me… from Laughing Through the Tears Blog When picking Wyatt up from school not long ago he got in my car and b…

Wishing everyone a happy and safe holiday weekend and sharing some beautiful photos from one of our talented patients.@ Carlos Pavia
https://l.facebook.com/l.php?u=https%3A%2F%2Fpaviaandleonephotography.godaddysites.com%2F%3Ffbclid%3DIwZXh0bgNhZW0CMTAAAR09dCvDJZfKtzynqn6BpjDUzCkb2MkNPu0l6RvmfkhcIJXQUijq5FBMczQ_aem_pU0Ytl9aSPyIWi2G3e8lxA&h=AT1iXR_iwftFNGjj_qrLPyCfGVYEkrrRXLj3Hd7dXfLYOW-mX0d-_UCE-LApbQAWGPPd4saFLcb8nVXEczdKPmrbULONpIPC95RIQJK5_MV9ZfpSmcNt3cIs9Ws6LaN3yzhBzauIOQ&__tn__=-UK-R&c[0]=AT1dcfsaewfiAmT_Y-tryTQKpzYW6LzY8crSEToD6Qm07Ym5tvQ2WQPNXWzkspFg5UHgGKVBtrXt6e-vTGFht7wKyy8Lryt1ernbROfCKnEDPERwWEIsAnHbF9D0fH4s4od7KiXo9lGXwpQDm1EKgqsbyv-vqg4Xld1XM4bWqY8abBChiUJo1DNGnWr4KCgdTz-UC-j8V9GSkvQOZ3wJ-DzWuA

Pavia & Leone Drone Photography We use cookies to analyze website traffic and optimize your website experience. By accepting our use of cookies, your data will be aggregated with all other user data.

Register by this Sunday, July 7, to save 30% on registration rates for PPMD's 2025 Annual Conference!
Mark your calendars! June 19-21, 2025, in Las Vegas as families, physicians, researchers, caregivers, industry partners, and those living with Duchenne, Becker, and carriers gather to connect, share information, and learn the latest progress in our fight for every future.Register by this Sunday, July 7, to save 30% on registration rates for PPMD's 2025 Annual Conference! register here:
https://web.cvent.com/event/ed1287ea-84b0-431c-ae98-ca0f0479d905/summary

https://content.govdelivery.com/accounts/USFDA/bulletins/3a50f34

FDA Guidance Provides New Details on Diversity Action Plans Required for Certain Clinical Studies FDA Guidance Provides New Details on Diversity Action Plans Required for Certain Clinical Studies U.S. Food and Drug Administration sent this bulletin at 06/26/2024 11:11 AM EDT If your email program has trouble displaying this email, view as a webpage. FDA Guidance Provides New Details on Diversity...

https://musculardystrophynews.com/dmd-exercise-and-physical-therapy/?fbclid=IwZXh0bgNhZW0CMTEAAR1SoeA-N-MC_9zRzUiHXeDCZwFyIDZElGiM6iIw_lt2uy59dySy2fhPecc_aem_Ew7uqDwmlCxYBk-qpkWB9w

Duchenne muscular dystrophy: Exercise and physical therapy –... Exercise and physical therapy can help a person with Duchenne muscular dystrophy maintain mobility, flexibility, and independence.

Register for CureDuchenne webinar tomorrow to learn more about Sareptas Gene Therapy and the recent expanded approval.

Welcome! You are invited to join a webinar: CureDuchenne Webinar: FDA Decision on Elevidys Explained. After registering, you will receive a confirmation email about joining the webinar. Join CureDuchenne for a webinar unpacking the recent FDA decision on Sarepta Therapeutics’ gene therapy, Elevidys, and what it means for the Duchenne community. Hear from Dr. Diana Castro, a board-certified neurologist and leading neuromuscular physician, who will share the latest information and ...

FDA press release regarding Sarepta GT expanded approval.

FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy The FDA expanded the approval of Elevidys, a gene therapy for the treatment of Duchenne muscular dystrophy for ambulatory and non-ambulatory individuals 4 years

Very good news!!!!

Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above | Sarepta Therapeutics, Inc. – FDA grants traditional approval to ELEVIDYS for ambulatory Duchenne patients – FDA grants accelerated approval to ELEVIDYS for non-ambulatory Duchenne patients – Sarepta will host an investor conference call on June 21, 2024 , at 8:30 a.m. ET CAMBRIDGE, Mass. --(BUSINESS WIRE)--Jun.

JKF Life-Enhancing Summer Retreat 2013 This video highlights our inaugural John Kerr Foundation Life-Enhancing Summer Retreat for young adults with Muscular Dystrophy. We had the honor of hosting 13 adults…