PO "Ukrainian parent project "MIO-LIFE"

24/10/2025

💊 Givinostat — A Difficult Decision for Duchenne Families

Joshua has been on Givinostat since July. Recently, there was an announcement about three reported deaths over the last 18 months in the Netherlands, Germany, and the USA involving boys on this treatment. Understandably, this has caused concern among Duchenne families everywhere.

The UK Duchenne consultants have released a statement confirming that the MHRA is urgently assessing these cases. At this time, there’s no clear evidence that the deaths were directly related to Givinostat, and other medical factors may have played a role.

For families like ours, deciding whether to continue treatment is incredibly hard. There are no easy answers — just a constant weighing of risks and hopes. Every parent must make the choice that feels right for their child, based on the information available and guidance from their care team.

Having Duchenne is heartbreaking. But as parents, we do everything we can to give our children a chance — a chance for more time, more comfort, more hope. 💛

Our hearts, thoughts, and prayers are with all the families affected. 🕊️

05/10/2025

👏👏👏

27/09/2025

https://www.facebook.com/reel/1119731257005194

21/09/2025

GIVINOSTAT EFFECT ON RESPIRATORY FUNCTION IN DUCHENNE MUSCULAR DYSTROP HY BEFORE AND AFTER AMBULATION LOSS: RESULTS FROM EPIDYS, LONG-TERM SAFETY AND TOLERABILITY

EXTENSION, AND PRO-DMD-01 STUDIES

(AANEM 2025 Meeting)

Craig M. McDonald (Sacramento, CA), Chamindra G.

Laverty (San Diego, CA), Erika L. Finanger (Portland,

OR), James E. Signorovitch (Boston, MA), Andrea Parodi

(Milan, Italy), Federica Alessi (Milan, Italy), Sara

Cazzaniga (Milan, Italy), Paolo Bettica (Milan, Italy), Yann

INTRODUCTION: Givinostat, an oral histone deacetylase

inhibitor, is indicated for the treatment of Duchenne

muscular dystrophy (DMD) in patients aged over sixyears-

old.

OBJECTIVE: To evaluate the effect of givinostat on

pulmonary function in patients who experienced loss of

ambulation (LoA) during follow-up.

METHODS: Data from the double-blind, randomized,

phase 3 EPIDYS study in ambulant boys (aged over sixyears-

old) with DMD (NCT02851797) and its ongoing

open-label extension (NCT03373968) were indirectly

compared with PRO-DMD-01 (NCT01753804), a natural

history study of DMD disease progression. Matchingadjusted

indirect comparisons were used, adjusting for

patient characteristics at LoA. Forced vital capacity (FVC)

%percent predicted mean trajectories before and after

LoA were estimated using longitudinal mixed effects

models.

RESULTS: This analysis included 56 patients treated with

givinostat and steroids compared with published data on

51 patients from the PRO-DMD-01 study who received

steroids only. Among weighted givinostat-treated patients,

two years before LoA, the weighted least squares mean

(SE) FVC %percent predicted was 91.3% (2.2%),

decreasing to 83.0% (2.3%) at LoA and 74.4% (2.4%) two

years post-LoA. The mean (SE) annual decline in FVC

percent predicted was 3.6% (1.2%) before LoA and 3.9%

(1.3%) after LoA. In the PRO-DMD-01 study, the mean

(SE) annual decline in FVC percent predicted was 5.6%

(2.1%) before LoA; this increased to 10.1% (2.2%) after

LoA.

SUMMARY/CONCLUSION: FVC% predicted trajectories

showed a slower and less pronounced decline in patients

treated with givinostat compared with those treated with

steroids only. These findings suggest improved pulmonary

function stabilization with givinostat treatment.

27/08/2025

🧬 Nuovi risultati sul trattamento con givinostat per la DMD

Italfarmaco ha pubblicato sulla rivista scientifica Annals of Clinical and Translational Neurology i dati di uno studio che ha seguito nel tempo i ragazzi con distrofia muscolare di Duchenne (DMD) trattati con givinostat.

I risultati mostrano che, in combinazione con i corticosteroidi, givinostat può rallentare la progressione della patologia nei pazienti deambulanti sopra i 6 anni.

In particolare:
🔹la perdita della capacità di alzarsi dal pavimento può essere ritardata di circa 2 anni
🔹la perdita della capacità di salire quattro gradini di circa 3,3 anni
🔹la perdita della deambulazione di circa 2,9 anni

📌 Lo studio conferma anche che il trattamento è stato generalmente ben tollerato, senza nuovi segnali di sicurezza.

🔗 Per leggere tutti i dettagli: https://www.parentproject.it/givinostat-pubblicati-i-dati-sul-trattamento-a-lungo-termine/

24/08/2025

СРОЧНЫЕ НОВОСТИ: Muscular Dystrophy Association и Parent Project Muscular Dystrophy выпустили новые Консенсусные рекомендации для безопасного и равноправного проведения генной терапии при мышечной дистрофии Дюшенна.

Эти рекомендации помогут группам по уходу ориентироваться на оценку, инфузию, контроль и последующую деятельность пациентов, гарантируя, что семьи будут иметь больше уверенности и поддержки при принятии решений по ухо

Читать полный выпуск ➡️ https://www.mda.org/press-releases/mda-and-ppmd-announce-joint-consensus-guidelines

BREAKING NEWS: Muscular Dystrophy Association and Parent Project Muscular Dystrophy have released new Consensus Guidelines for the safe and equitable delivery of gene therapy in Duchenne muscular dystrophy.

These recommendations will help care teams navigate patient evaluation, infusion, monitoring, and follow-up, ensuring that families have more confidence and support in their care decisions.

Read the full release ➡️ https://www.mda.org/press-releases/mda-and-ppmd-announce-joint-consensus-guidelines

18/08/2025

https://www.globenewswire.com/news-release/2025/07/11/3113828/0/en/Italfarmaco-Presents-New-Cardiac-Data-for-Givinostat-at-16th-European-Paediatric-Neurology-Society-EPNS-Congress.html?fbclid=IwQ0xDSwMP3WJjbGNrAw_dM2V4dG4DYWVtAjExAAEeQKhIITYshX7G8TtsyQFQaa4QNSrR0BskdgDJC_2geSlgpht3lAKfybRnL7k_aem_RRp5mx8Df8jYZdvJY7KeGA

In total, 11 abstracts on givinostat were accepted for presentation at the Congress, including two for an oral presentation MILAN, Italy, July...

27/07/2025

08/07/2025