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Announcing: Special Issue | Volume 8, Issue 5

The Null Hypothesis Stands

Guest Editors: Annemieke Aartsma-Rus and Virginia Arechavala-Gomeza

Extract of the editorial by the guest editors: "It is clear that results that prove an original hypothesis to be incorrect advance knowledge as much as those that confirm it. Still, in the era of 'publish or perish' results that disprove the hypothesis tested are not published at the rate that should be expected, and the lack of reproducibility has also been recently highlighted as a problem that needs to be addressed. A publication bias towards therapeutic approaches that appear to have beneficial effects and lack of published studies where therapies are either not useful or toxic, or where published results are not reproducible, not only impedes science and therapy development. It also leads to unnecessary duplication of efforts and a waste of resources. In any field this is not optimal, but in a rare disease field – such as research in neuromuscular disorders – this is a tragedy. In light of this, we are happy to present a whole issue dedicated to well-executed research that obtained what many may consider 'negative' results."

Most Read JND Articles in August 2021

Listing articles published only in 2020 and 2021 so you can read the most popular recent content

Intrahepatic Cholestasis Is a Clinically Significant Feature Associated with Natural History of X-Linked Myotubular Myopathy (XLMTM): A Case Series and Biopsy Report – Open Access (Research Article in pre-press, 2021)
Molera, Cristina | Sarishvili, Tinatin | Nascimento, Andrés | Rtskhiladze, Irakli | Muñoz Bartolo, Gema | Fernández Cebrián, Santiago | Valverde Fernández, Justo | et al.

Molecular Mechanisms of Skeletal Muscle Hypertrophy – Open Access (Review Article in Vol.8, Iss.2, 2021)
Schiaffino, Stefano | Reggiani, Carlo | Akimoto, Takayuki | Blaauw, Bert

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment – Open Access (Research Article in pre-press, 2021)
Mitelman, Olga | Abdel-Hamid, Hoda Z. | Byrne, Barry J. | Connolly, Anne M. | Heydemann, Peter | Proud, Crystal | Shieh, Perry B. | Wagner, Kathryn R. | et al.

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial – Open Access (Research Article in pre-press, 2021)
McDonald, Craig M. | Shieh, Perry B. | Abdel-Hamid, Hoda Z. | Connolly, Anne M. | Ciafaloni, Emma | Wagner, Kathryn R. | Goemans, Nathalie | Mercuri, Eugenio | et al.

Current View of Diagnosing Small Fiber Neuropathy – Open Access (Review Article in Vol.8, Iss.2, 2021)
Raasing, Lisette R.M. | Vogels, Oscar J.M. | Veltkamp, Marcel | van Swol, Christiaan F.P. | Grutters, Jan C.

Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care – Open Access (Review Article in Vol.7, Iss.1, 2020)
Schorling, David C. | Pechmann, Astrid | Kirschner, Janbernd