CureDuchenne

CureDuchenne is pleased to share that PTC Therapeutics has announced that the FDA has accepted for review the New Drug Application (NDA) for Translarna (ataluren) for the treatment of Duchenne caused by nonsense mutations. The resubmission includes results from a global placebo-controlled study showing significant functional benefits at 72 weeks of treatment, as well as significant long-term benefits captured in the STRIDE registry. See the press release and letter to the community: https://lnkd.in/gKTtuCH7 #FDA #DMD #Duchenne #CureDuchenne #PTC

Jeg har fått en del spørsmål om hvordan filmen om Ibelin har blitt mottatt de første dagene etter at den ble lagt ut på Netflix fredag. Det har vært fullstendig overhvelmende. I World of Warcraft har hundrevis av spillere besøkt graven til Ibelin, tent lys og delt refleksjoner og minner. Det er en stadig strøm av spillere som kommer til graven og det rapporteres at det har vært avatarer ved graven 24/7 siden fredag. #Fellesskap og #kjærlighet. Vi får en stadig strøm av meldinger fra personer som har sett filmen. Fra Brisbane (Australia) til San Fransisco (USA) med Asia og Europa i midten. Mennesker som deler både sine opplevelser og situasjoner i egne liv. #Åpenhet og #inkludering. Enkeltpersoner legger ut små videosnutter på YouTube og sosiale medier av hvordan de ser ut når de ser filmen. Og ulike mediekanaler over hele verden skriver om opplevelsen slik som Aftenposten gjorde i Norge i dag. #Tårerforhåp. Når det gjelder seertall på Netflix så gikk Ibelin til topps som den mest sette film i Norge allerede første dagen og den har ligget der siden. I Sverige, Danmark og Finland er den den nest mest sette filmen etter bare fire dager og i Island nr3. I tillegg er den på topp 10 listen i Tyskland, Belgia, Estland, Latvia og Sveits - etter kun fire dager. Dette blir spennende å følge i dagene og ukene som kommer. #Respekt. I dag kunngjorde også organisasjonen «Cinema Eye» de 16 nominerte filmene til årets publikumspris i verden. Prisen er en av de mest attraktive publikumspriser som deles ut i verden. Norge har aldri vunnet denne prisen og i år er «The Remarkable Life of Ibelin» en av de 16 nominerte(!). Det er filmpublikum over hele verden (du og jeg) som stemmer fram vinnerne som blir kunngjort i New York 9. januar 2025. Første globale avstemningsrunde varer fra i dag og til og med lørdag. Alle kan stemme og i fjor vant filmen «People’s President» med litt over 150.000 stemmer. Skulle du ha lyst til å støtte Ibelin i konkurransen om årets publikumspris og derigjennom gjøre filmen enda mer kjent for publikum over hele verden så KAN du det ved å klikke deg inn på linken under og stemme på «The Remarkable Life of Ibelin». Du kan også dele denne posten med venner og bekjente. Jeg skal holde dere oppdatert om resultatene. #Fellesskap og #Ibelin 🦊.

We have profound gratitude to Robert Steen for sharing his story, Mats’ journey as “Ibelin” in World of Warcraft®, and for helping raise awareness about Duchenne and CureDuchenne. To learn more, watch the documentary, and get the Reven Pack in World of Warcraft click here: https://lnkd.in/gpxVvRzH #Ibelin #WorldOfWarcraft #DMD #Netflix #Duchenne #CureDuchenne #WOW

Thank you @Hawken for sharing! This is a great read!!

🎥 We're excited to share the story of The Remarkable Life of Ibelin, the inspiring Netflix documentary about Mats Steen, a young man with Duchenne who found community, friendship, and purpose through World of Warcraft®. A heartfelt thank you to the Steen family for sharing Mats' powerful story, reminding us of the strength and connection that can come from unexpected places. 🙏 We're also incredibly grateful to Blizzard Entertainment for their generous support of CureDuchenne's mission. You can honor Mats’ legacy by adopting Reven, a limited-edition pet fox in World of Warcraft®, in support of CureDuchenne to accelerate a cure for Duchenne and provide resources and support for families living with Duchenne. 🦊💙 Learn more, watch the documentary, and get the Reven Pack in World of Warcraft®: https://lnkd.in/gpxVvRzH #Duchenne #Ibelin #CureDuchenne #DMD #RevenPack #WoW #WorldofWarcraft #netflix

Pfizer recently provided this video updating the results from their Phase 3 micro-dystrophin gene therapy trial, CIFFREO, which were presented on October 12, 2024 at the World Muscle Society Conference in Prague. In this video, Francesco Muntoni, Chair of the CIFFREO trial steering committee, and Dan Levy, who leads DMD Clinical Research at Pfizer review the data--which showed no statistically significant improvements in individuals receiving the experimental micro-dystrophin gene therapy--and have a thoughtful discussion about the implications of the findings. While we’re disappointed with the results of the trial, we appreciate Pfizer’s continued transparency to share as much information as possible with the whole community. Watch the full video: https://lnkd.in/gNvXftuZ #DMD #GeneTherapy #FDA #duchenne #CureDuchenne #musculardystrophy

World Mental Health Day serves as a crucial reminder for everyone to prioritize emotional well-being, especially within communities affected by challenging conditions like Duchenne muscular dystrophy. In a CureDuchenne webinar, pediatric psychologist Dr. Paige Lembeck offered valuable strategies to manage stress, anxiety, and depression for both individuals with Duchenne and their caregivers. Stress is an inevitable part of life, but understanding how to recognize the signs and manage emotional health effectively can make all the difference. Dive into our blog for expert tips and resources to support mental wellness in your journey with Duchenne. #mentalhealth #mentalhealthmatters #mentalhealthawareness #DMD #duchenne #CureDuchenne https://lnkd.in/gXjTASit

CureDuchenne is proud to champion newborn screening for Duchenne muscular dystrophy through initiatives like our program at Brigham and Women’s Hospital in Massachusetts, as well as through ongoing advocacy efforts across the nation. With new treatments now available, newborn screening is crucial in identifying children with Duchenne early, giving them the opportunity to begin treatments between the ages of 0 and 4. Without newborn screening, many children remain undiagnosed during this critical window, missing the chance to preserve muscle strength and delay the progression of the disease. #DMD #CureDuchenne #Duchenne #research #FDA #newbornscreening

We’re at the 29th Annual Congress of the World Muscle Society this week and are pleased to share updates from companies presenting on the latest advancements in Duchenne muscular dystrophy treatments. Here are the latest press releases: https://lnkd.in/gxXXYt5v

10 years ago, CureDuchenne provided seed funding to #cTAP, and we are proud to see the unanimous support this initiative has received from the Duchenne community. Thank you Susan Ward for your vision and tenacity in creating such a useful tool. cTAP's most recent study, using natural history data provided by #CureDuchenne, suggests trial enrollment criteria should expand beyond whether or not a patient can walk. Read more about this study and perspectives from families here: https://lnkd.in/eVDi5pfa. Rare Disease Advisor