Duchenne muscular dystrophy (DMD) is an X-linked genetic disease, caused by the absence of the dystrophin protein. Although many novel therapies are under development for DMD, there is currently no cure and affected individuals are often confined to a wheelchair by their teens and die in their twenties/thirties. DMD is a rare disease (prevalence <5/10,000). Even the largest countries do not have enough affected patients to rigorously assess novel therapies, unravel genetic complexities, and determine patient outcomes. TREAT-NMD is a worldwide network for neuromuscular diseases that provides an infrastructure to support the delivery of promising new therapies for patients. The harmonized implementation of national and ultimately global patient registries has been central to the success of TREAT-NMD. For the DMD registries within TREAT-NMD, individual countries have chosen to collect patient information in the form of standardized patient registries to increase the overall patient population on which clinical outcomes and new technologies can be assessed. The registries comprise more than 13,500 patients from 31 different countries. Here, we describe how the TREAT-NMD national patient registries for DMD were established. We look at their continued growth and assess how successful they have been at fostering collaboration between academia, patient organizations, and industry.

Abstract. We present an axisymmetric numerical model of a dynamo active accretion disc. If the dynamo-generated magnetic field in the disc is sufficiently strong (close to equipartition with thermal energy), a fast magneto-centrifugally driven outflow develops within a conical shell near the rotation axis, together with a slower pressure driven outflow from the outer parts of the disc as well as around the axis. Our results show that a dynamo active accretion disc can contribute to driving an outflow even without any external magnetic field. The fast outflow in the conical shell is confined by the azimuthal field which is produced by the dynamo in the disc and advected to the disc corona. This part of the outflow has high angular momentum and is cooler and less dense than its surroundings. The conical shell's half-opening angle is typically about 30• near the disc and decreases slightly with height. The slow outflow is hotter and denser.

Abstract.The interaction between a protostellar magnetosphere and a surrounding dynamo-active accretion disc is investigated using an axisymmetric mean-field model. In all models investigated, the dynamo-generated magnetic field in the disc arranges itself such that in the corona, the field threading the disc is anti-aligned with the central dipole so that no X-point forms. When the magnetospheric field is strong enough (stellar surface field strength around 2 kG or larger), accretion happens in a recurrent fashion with periods of around 15 to 30 days, which is somewhat longer than the stellar rotation period of around 10 days. In the case of a stellar surface field strength of at least a few 100 G, the star is being spun up by the magnetic torque exerted on the star. The stellar accretion rates are always reduced by the presence of a magnetosphere which tends to divert a much larger fraction of the disc material into the wind. Both, a pressure-driven stellar wind and a disc wind form. In all our models with disc dynamo, the disc wind is structured and driven by magneto-centrifugal as well as pressure forces.

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Abstract.We consider the effect of vertical outflows on the mean-field dynamo in a thin disk. These outflows could be due to winds or magnetic buoyancy. We analyse both two-dimensional finite-difference numerical solutions of the axisymmetric dynamo equations and a free-decay mode expansion using the thin-disk approximation. Contrary to expectations, a vertical velocity can enhance dynamo action, provided the velocity is not too strong. In the nonlinear regime this can lead to super-exponential growth of the magnetic field.