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1655 news items
Research
A good record for the longest follow-up to date of Zolgensma in SMA
On 03/06/2021
After obtaining positive results on the motor development of 15 infants with type I SMA, aged less than 6 months and symptomatic during the first…
Research
A single injection of a promising double gene therapy in a OPMD mouse model
On 03/06/2021
Oculopharyngeal muscular dystrophy (or OPMD) is a relatively rare type of muscular dystrophy that tends to affect and older population. This condition is transmitted in…
Research
DMD : a robust production of dystrophin, 3 months after administration of SRP-9001 in 11 patients (phase I clinical trial)
On 03/06/2021
SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) developed in Duchenne muscular dystrophy (DMD) by Sarepta Therapeutics is a gene therapy product combining a micro-dystrophin gene and an adeno-associated virus (AAV).…
Events
M&M’s – Muscle Monday Seminar – 7 June – Tom Cooper (USA)
On 01/06/2021
Mechanistic insights from modeling cardiac features of Myotonic Dystrophy Type 1 in mice Monday June 7th 2021 from 3pm to 4 pm Tom Cooper (Professor, Baylor…
Research
SMA: results of a large-scale survey of newborn screening around the world
On 31/05/2021
The question of neonatal screening for SMA does rise more and more since the marketing of three disease-modifying therapies, Spinraza®, Zolgensma® and Evrysdi®, which efficacy…
Research
FSHD: a new european network for clinical trials
On 31/05/2021
On the initiative of FSHD Europe association, in which the AFM-Telethon is involved, a European network of clinical trials in FSH has just been created…
Events
The Summer School of Myology is changing, becoming AcadeMYO – Interview with Norma B. Romero and J. Andoni Urtizberea
On 27/05/2021
The Institute’s Summer School of Myology becomes AcadeMYO this year. Entirely virtual, it will take place from July 5th to 9th, in partnership with the…
Research
Motor neurons and myotubes interact via secreted endogenous neuronal and muscle growth factors
On 25/05/2021
Neuromuscular junction (NMJ) research is essential to advance the understanding of neuromuscular pathophysiology and the development of new therapies for diseases associated with NMJ dysfunction.…
Events
ERN EURO-NMD webinar, 27 May: Prof. Sabrina Sacconi and Dr Teresinha Evangelista
On 25/05/2021
Webinar organised by EURO-NMD in collaboration with ERN-RND and EAN. Thursday 27 mai 2021 – 4:00 am (Paris time) What’s new in FSHD…
Events
M&M’s – Muscle Monday Seminar – 31 May – Ronenn Roubenoff
On 25/05/2021
Leave the guns, take the cannoli: What bimagrumab tells us about the myostatin pathway in humans Monday May 31st 2021 from 12 am to 1 pm…
Research
Is the risk-taking inherent in DMD gene therapy acceptable to patients?
On 25/05/2021
Duchenne muscular dystrophy (DMD), the most common form of myopathy in children, is currently experiencing major developments in innovative therapies. This neuromuscular disease leading to…
Research
A heterozygous variant of the calpain 3 gene causing camptocorm syndrome
On 21/05/2021
Calpainopathies are a subgroup of limb girdle muscular dystrophies whose symptoms usually appear in early adolescence. In the vast majority of cases, this neuromuscular disease,…
Research
Reprogrammed pluripotent stem cells: formidable tools to test innovative neuromuscular disease therapies
On 20/05/2021
In the field of innovative therapies for neuromuscular diseases, having access to a model, whether cell or animal, for a given disease is a major…
Events
The Institute is recruiting a Webmaster & Community manager (M/F) for EURO-NMD
On 20/05/2021
The Institute of Myology Located in Paris at the heart of Europe’s largest hospital, the Pitié-Salpêtrière, the Institute of Myology was created in 1996 under…
Research
Type II and III SMA: positive effects of nusinersen, greater impact on motor function than on respiratory function
On 19/05/2021
Of the many forms of spinal muscular atrophy (SMA), the form causing predominately proximal muscle deficit and associated with a lack of the SMN protein…
Events
French-Iranian symposium on Therapeutic Updates in Neuromuscular Disorders – May 21st – 12:30
On 17/05/2021
The French-Iranian symposium, supported by FILNEMUS (the French Rare Health Care for Neuromuscular Diseases Network), will take place by videoconference on Friday, May 21st, 2021,…
Research
Using gene therapy to treat CMT 1A through RNA interference
On 14/05/2021
Charcot-Marie-Tooth disease type 1A (CMT1A) is an inherited sensory-motor neuropathy linked to the duplication of the PMP22 gene. Excessive production of PMP22 by Schwann cells…
Events
Launch of ENMC Mid-Career Mentoring Programme – Interview with Dr Ana Ferreiro
On 11/05/2021
The European Neuromuscular Centre (ENMC) is launching a mentoring programme this year aimed at mid-career neuromuscular disease specialists. This programme is open to doctors and…
Research
A low level of anti-AAV9 antibodies makes most very young patients with SMA eligible for Zolgensma
On 10/05/2021
Pre-existing immunity against adeno-associated viruses (AAVs), which are naturally very widespread, is one of the obstacles to gene therapy using such a vector. Zolgensma (onasemnogene…
Research
Treating cardiomyopathy earlier with angiotensin converting enzyme inhibitors preserves heart function in BMD
On 10/05/2021
Becker muscular dystrophy (BMD) is the result of a partial dystrophin deficiency. Approximately 40% of adults with BMD present dilated cardiomyopathy, including 15% of under…