3 - Specific competencies and capacity to provide export advice
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Quality
Management |
Training and
Education |
Public
Involvement |
| Description | ||
|---|---|---|
Name |
TREAT-NMD - Neuromuscular Network |
|
| Number of peer reviewed publications in the last 5 years | 5 | 1. Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA. Hoffman EP; Workshop Participants.; TREAT-NMD Alliance.Neuromuscul Disord. 2017 Mar 4. pii: S0960-8966(16)31100-2. doi: 10.1016/j.nmd.2017.02.013 2. Compliance to Care Guidelines for Duchenne Muscular Dystrophy. Compliance to Care Guidelines for Duchenne Muscular Dystrophy. Landfeldt E, Lindgren P, Bell CF, Schmitt C, Guglieri M, Straub V, Lochmüller H, Bushby K. J Neuromuscul Dis. 2015;2(1):63-72. 3. The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. Heslop E, Csimma C, Straub V, McCall J, Nagaraju K, Wagner KR, Caizergues D, Korinthenberg R, Flanigan KM, Kaufmann P, McNeil E, Mendell J, Hesterlee S, Wells DJ, Bushby K; TACT.. Orphanet J Rare Dis. 2015 Apr 23;10:49. doi: 10.1186/s13023-015-0258-1 4. The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations. Bladen CL, et al. Hum Mutat. 2015 Apr;36(4):395-402. doi: 10.1002/humu.22758. 5. The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseases. Rodger S, Lochmüller H, Tassoni A, Gramsch K, König K, Bushby K, Straub V, Korinthenberg R, Kirschner J. Orphanet J Rare Dis. 2013 Oct 23;8:171. doi: 10.1186/1750-1172-8-171 |
| Number of competitive grants obtained in the last 5 years | 0 | Publications have involved multiple members of the TREAT-NMD membership as well as the TREAT-NMD Executive Committee and senior management team. |
| Access to expert groups | Yes | The TACT (TREAT-NMD Advisory Committee for Therapeutics) is an established expert group who meet twice a year to review and provide guidance on the translation and development path of therapeutics programs in rare neuromuscular diseases |
| Capacity to answer external scientific questions | Yes | The TACT (TREAT-NMD Advisory Committee for Therapeutics) is an established expert group who meet twice a year to review and provide guidance on the translation and development path of therapeutics programs in rare neuromuscular diseases |
| Site feasibility | No | |
| Participant recruitment | No | |
| Budget calculation for studies | No |