Blog Archives
SMA : start of DEVOTE clinical trial in the US
The DEVOTE trial is a phase II / III randomized, controlled, dose escalation trial that aims to assess the tolerance and efficacy of a higher dose of nusinersen (Spinraza®) in SMA than the one currently used. It will take place in nearly 50 countries, including in France. Biogen announced in a press release dated April … [Read more]
Proof of concept of a plasmapheresis specifically filtering AAVs
Neutralizing antibodies directed against adeno-associated virus (AAV) are commonly found in humans. In seropositive subjects, vector administration is not feasible as antibodies neutralize AAV vectors even at low titers. Consequently, a relatively large proportion of humans is excluded from enrollment in clinical trials and, similarly, vector redosing is not feasible because of development of high-titer … [Read more]
COVID-19 and clinics: 4 questions for Dr Guillaume Bassez
Interview with Guillaume Bassez, research professor and neurologist at the Institute of Myology’s Service of Neuro-Myology. What measures have been put in place at the Service of Neuromyology to respond to the COVID-19 health crisis? Our service comprises 10 doctors: neurologists, rehabilitation doctors, psychologists and two interns. We usually handle 4200 consultations per year and … [Read more]
CHMP issues a positive opinion on Zolgensma® in SMA
Zolgensma®, initially developed by AveXis (subsequently acquired by Novartis) has just received a positive opinion in SMA by the by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The CHMP recommends a conditional marketing authorization which should only concern “babies and young children”: suffering from proximal spinal muscular … [Read more]
Myasthenia gravis reduces the chances of being able to work, especially in its refractory form
Autoimmune myasthenia gravis (MG), a dysimmunity disorder of the neuromuscular junction, begins in 60% of cases in an adult before the age of 40, that is, in full “active life”. So what is its impact on the possibility of exercising a professional activity? Two publications provide answers: MG would reduce the employment rate by 15 … [Read more]
COVID-19 and clinical trials: 4 questions for Dr Giorgia Querin
Interview with Giorgia Querin, neurologist and Coordinating Doctor for the adult clinical trials platform at the Institute of Myology since January 2020. What measures have been put in place at the Service of Neuro-Myology and at the adult clinical trials platform in response to the COVID-19 health crisis? We have reviewed our organisation to … [Read more]
Losmapimod is well tolerated in FSH type 1
Losmapimod is a p38 MAP kinase inhibitor, which has already shown good tolerance in several diseases (myocardial infarction, chronic obstructive pulmonary disease, etc.). In FSH, encouraging results in vitro and in vivo showing that losmapimod reduces the expression of the DUX4 gene have confirmed the launch of clinical trials by the company Fulcrum Therapeutics. The … [Read more]
Genetic abnormalities involved in SMALED2 better understood
Spinal muscular atrophy, lower extremity-predominant type 2, or SMALED2 is a rare form of proximal spinal muscular atrophy. It usually progresses more slowly than SMA. Two forms can be found: SMALED 2A, the classic form of the disease, begins in childhood and evolves very slowly, and SMALED 2B, more severe, whose first manifestations appear in … [Read more]
DMD: a drug targeting exon-skipping of exon 53 authorized in Japan
The Japanese laboratory Shinyaku Co. Ltd announced on March 25, 2020 by press release the commercial approval in Japan of its antisense oligonucleotide targeting exon-skipping of exon 53 of the dystrophin gene (DMD gene), viltolarsen (NS- 065 / NCNP-01- Viltepso), in Duchenne muscular dystrophy. Viltolarsen 250 mg is given by intravenous injection, once a week, … [Read more]
Two Cochrane reviews conclude that nusinersen is effective in SMA type I and II
Dutch and American resarchers have published an update to October 2018 of two Cochrane reviews concerning clinical trials of treatments able to stop or slow the progression of SMA type I, II or III. Previous editions (in 2009 and 2011) had not highlighted effective treatments in SMA; since then, nusinersen has been tested and has … [Read more]
