With @worldmusclesociety.bsky.social royalty E/Prof Victor Dubowitz joined us for the programme committee dinner. Wonderful to see Victor again and in such great spirits. #WMS2025 #myology #peripateticpaediatrician #neuromusculardiseases
May 22, 2025 at 7:04 AM
With @worldmusclesociety.bsky.social royalty E/Prof Victor Dubowitz joined us for the programme committee dinner. Wonderful to see Victor again and in such great spirits. #WMS2025 #myology #peripateticpaediatrician #neuromusculardiseases
#chronicillness #genericdisorders #rarediseases #neuromusculardiseases #musculardystrophy #geneticmutation
People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid recent advancements in the growing field of genetic science.
Genetic medicine can leave people with rare mutations behind. But there's new hope
People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid recent advancements in the growing field of genetic science.
bit.ly
April 27, 2025 at 8:12 AM
Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#MDA #SonoThera #DMD #neuromusculardiseases #nonviral #ultrasound #genetherapy
#MDA #SonoThera #DMD #neuromusculardiseases #nonviral #ultrasound #genetherapy
March 5, 2026 at 7:10 PM
Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#MDA #SonoThera #DMD #neuromusculardiseases #nonviral #ultrasound #genetherapy
#MDA #SonoThera #DMD #neuromusculardiseases #nonviral #ultrasound #genetherapy
#PCBCommunity | A team from #SJDbarcelona Hospital Research Institute ( #IRSJD), and #CNAG, based in the #BarcelonaSciencePark, have contributed to the diagnosis of 23 children with #neuromusculardiseases, ending a diagnostic odyssey that, in some cases, lasted more than 8 years.
👉 bit.ly/3ZTleVr
👉 bit.ly/3ZTleVr
An innovative genetic analysis by SJD Barcelona Children’s Hospital and CNAG successfully diagnoses 23 children with neuromuscular diseases
A team of researchers from the Sant Joan de Déu Research Institute (IRSJD) and the Centro Nacional de Análisis Genómico (CNAG), based in the Barcelona Science Park, have contributed to the diagnosis o...
bit.ly
December 17, 2024 at 1:21 PM
#PCBCommunity | A team from #SJDbarcelona Hospital Research Institute ( #IRSJD), and #CNAG, based in the #BarcelonaSciencePark, have contributed to the diagnosis of 23 children with #neuromusculardiseases, ending a diagnostic odyssey that, in some cases, lasted more than 8 years.
👉 bit.ly/3ZTleVr
👉 bit.ly/3ZTleVr
Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#neuromusculardiseases #MDA #genetherapy #nonviral #ultrasound #DMD #SonoThera
#neuromusculardiseases #MDA #genetherapy #nonviral #ultrasound #DMD #SonoThera
March 5, 2026 at 7:07 PM
Pleased to be sharing our latest data with the DMD community at MDA 2026 highlighting RIPPLE™ as an efficient, scalable, nonviral platform for full-length dystrophin delivery in DMD. Get more details: www.sonothera.com
#neuromusculardiseases #MDA #genetherapy #nonviral #ultrasound #DMD #SonoThera
#neuromusculardiseases #MDA #genetherapy #nonviral #ultrasound #DMD #SonoThera
#generalisedmyastheniagravis #gMG #UCB #Rystiggo #rozanolixizumab #NICE #NHS #HEOR #Neuromusculardiseases #uncontrolledgMGtherapy #rarediseases #NICEguidance #reimbursement #NICEcosteffectivenessmodelling #NICEdecisionmaking #InnovativeMedicinesFund #IMF #gMGcommunitypatients
zurl.co/Tj9mI
zurl.co/Tj9mI
NICE backs UCB drug as first uncontrolled gMG therapy
UCB's Rystiggo can be used by the NHS as a backup treatment for people with the rare autoimmune and neuromuscular disease gMG, says NICE.
pharmaphorum.com
May 6, 2026 at 9:38 AM
neuromuscular-diseases-news.webnode.page/
#NEUROMUSCULARDISEASES
#NEUROMUSCULARDISEASES
August 27, 2025 at 10:28 AM
neuromuscular-diseases-news.webnode.page/
#NEUROMUSCULARDISEASES
#NEUROMUSCULARDISEASES
Bone health in neuromuscular diseases is an important topic.
This paper highlights the various aspects and is intended to sensitize patients and healthcare professionals to this subject.
www.jns-journal.com/article/S002...
#neuromusculardiseases #rarediseases
This paper highlights the various aspects and is intended to sensitize patients and healthcare professionals to this subject.
www.jns-journal.com/article/S002...
#neuromusculardiseases #rarediseases
Awareness of bone strength in patients with neuromuscular disorders: ERN EURO-NMD clinician survey and European patient survey
Bone strength is reduced In various neuromuscular disorders (NMDs). We aimed to assess
the awareness and practice of bone strength management in NDMs among clinicians and
patients. We performed two on...
www.jns-journal.com
February 25, 2025 at 4:03 PM
Bone health in neuromuscular diseases is an important topic.
This paper highlights the various aspects and is intended to sensitize patients and healthcare professionals to this subject.
www.jns-journal.com/article/S002...
#neuromusculardiseases #rarediseases
This paper highlights the various aspects and is intended to sensitize patients and healthcare professionals to this subject.
www.jns-journal.com/article/S002...
#neuromusculardiseases #rarediseases