A National Spinal Muscular Atrophy Registry for Real-World Evidence.

BACKGROUND: Spinal muscular atrophy (SMA) is a devastating rare disease that affects individuals regardless of ethnicity, gender, and age. The first-approved disease-modifying therapy for SMA, nusinursen, was approved by Health Canada, as well as by American and European regulatory agencies following positive clinical trial outcomes. The trials were conducted in a narrow pediatric population defined by age, severity, and genotype. Broad approval of therapy necessitates close follow-up of potential rare adverse events and effectiveness in the larger real-world population. METHODS: The Canadian Neuromuscular Disease Registry (CNDR) undertook an iterative multi-stakeholder process to expand the existing SMA dataset to capture items relevant to patient outcomes in a post-marketing environment. The CNDR SMA expanded registry is a longitudinal, prospective, observational study of patients with SMA in Canada designed to evaluate the safety and effectiveness of novel therapies and provide practical information unattainable in trials. RESULTS: The consensus expanded dataset includes items that address therapy effectiveness and safety and is collected in a multicenter, prospective, observational study, including SMA patients regardless of therapeutic status. The expanded dataset is aligned with global datasets to facilitate collaboration. Additionally, consensus dataset development aimed to standardize appropriate outcome measures across the network and broader Canadian community. Prospective outcome studies, data use, and analyses are independent of the funding partner. CONCLUSION: Prospective outcome data collected will provide results on safety and effectiveness in a post-therapy approval era. These data are essential to inform improvements in care and access to therapy for all SMA patients

Preliminary study on the ecomorphological signification of the sound-producing complex in Carapidae

Carapidae can be classified in four ecological groups : pelagic, dermersal, commensal and parasitic. Carapidae display otophysic structures associated with the anterior part of the swim bladder and highly modified labyrinths, which suggest particular acoustic performances. The commensal and parasitic species have the best developed sound-producing features and also the thickest sagitta within the largest otic cavity, and surrounded by the thinnest cranial wall. However, these features do not necessarily imply a direct relation between the sound emission and reception in a given species but suggest a selective pressure lying in the habitat use of the species. The structures involved in sound-production and hearing are seemingly adapted to match the loss of energy of the sonic vibrations when travelling through the host tissues

The branchial basket in Teleost feeding.

In teleosts, feeding is effected principally by suction and food is handled by the branchial basket. Preys are carried to the oesophagus by the pharyngeal jaws (PJs). The pharyngobranchial bones constitute the upper pharyngeal jaws (UPJs) and the 5th ceratobranchial bones, the lower pharyngeal jaws (LPJs). In lower teleosts, these jaws have well-separated spindly parts attached to the neurocranium, pectoral girdle, and hyoid bar; they only transport food and LPJ activity predominates. In acanthopterygians, the PJs become stronger, the left and right ceratobranchials fuse into one LPJ, and the pharyngobranchials join together to form two big UPJs articulating with the neurocranium. In labrids and scarids, the LPJ is also joined to the pectoral girdle. In acanthopterygians, a new retractor dorsalis muscle gives the UPJs the major role in food chewing and transport. Cypriniforms have developed original PJs with strong 5th ceratobranchials opposed to a postero-ventral neurocranial plate. Small-sized preys and food particles are seized by the gill rakers, small skeletal pieces supported by the branchial arches

T. Muxart et P. Birot. — L'altération météorique des roches.

Chardon Michel. T. Muxart et P. Birot. — L'altération météorique des roches.. In: Revue de géographie alpine, tome 66, n°1, 1978. pp. 102-103

Bravard J.P. — Le Rhône du Léman à Lyon.

Chardon Michel. Bravard J.P. — Le Rhône du Léman à Lyon.. In: Revue de géographie alpine, tome 75, n°1, 1987. pp. 89-91

Géomorphologie et risques naturels : l'éboulement de Séchilienne (Isère) et ses enseignements

Abstract. — For some years, in Romanche valley (Isère, France) Séchilienne débris talus has an active evolution and a large landslide is possible. Morphological features and processus are described : mainly numerous fractures in relation with structure, déglaciation and occasionnaly old mining activities explain suffosion phenomena in the upper part. Frost action and rain wash are the main processus generating rock-dribbles, rock-slides and débris flows. Similar and dangerous phenomena are now spreading over the next slopes in this valley.Résumé. — L'éboulement de Séchilienne (vallée de la Romanche, Isère) prend depuis quelques années une ampleur catastrophique et fait peser une menace sur la vallée. Les conditions morphologiques sont analysées, les processus d'évolution sont décrits : la dislocation des roches crée des phénomènes de suffosion, l'action du gel/dégel et le ravinement sont à l'origine des chutes de pierres et de blocs. Des phénomènes similaires et préoccupants se développent sur les versants voisins.Chardon Michel. Géomorphologie et risques naturels : l'éboulement de Séchilienne (Isère) et ses enseignements. In: Revue de géographie alpine, tome 75, n°3, 1987. pp. 249-261

Livres reçus

Chardon Michel. Livres reçus. In: Revue de géographie alpine, tome 81, n°4, 1993. pp. 101-103

Rossi (G.), 1980. — L'extrême-nord de Madagascar.

Chardon Michel. Rossi (G.), 1980. — L'extrême-nord de Madagascar. . In: Revue de géographie alpine, tome 69, n°3, 1981. pp. 510-512

A. Godard. — Pays et paysages du granite.

Chardon Michel. A. Godard. — Pays et paysages du granite. . In: Revue de géographie alpine, tome 66, n°1, 1978. pp. 97-99

Tills and related deposits - Genesis, Petrology, Application, stratigraphy (1983).

Chardon Michel. Tills and related deposits - Genesis, Petrology, Application, stratigraphy (1983). In: Revue de géographie alpine, tome 72, n°1, 1984. p. 123