viernes, 20 de septiembre de 2019

Orphan Disease Center | Home

Orphan Disease Center | Home



PATIENTS ADVANCING RESEARCH


To Apply: 

LOI DUE MONDAY, SEPTEMBER 23, 2019 by 8pm EST

  1. Review the 2019 MDBR RFA Guidelines.

  2. Complete the LOI Form on Submittable 

Please note: If you are new to submittable it will ask you sign up as a new user before you can submit your application.


Research Areas for the 2019 MDBR Grant Program:


  1. Adult Polyglucosan Body Disease (APBD)
  2. Ataxia-Telangiectasia (A-T)
  3. BPAN- A Neurodegeneration with Brain Iron Accumulation Disorder
  4. CADASIL: Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy 
  5. Castleman Disease
  6. CDKL5 Deficiency Disorder
  7. Charcot Marie Tooth (CMT)
  8. Cystic Fibrosis
  9. Choroideremia (CHM)
  10. Congenital Hyperinsulinism (CHI)
  11. Congenital Muscular Dystrophy (CMD)
  12. CRB1 (Retinal Blindness)
  13. Dyskeratosis Congenita & Telomere Biology Disorder
  14. Fibrous Dysplasia (FD)
  15. Fibrodysplasia Ossificans Progressiva (FOP)
  16. Generalized Lymphatic Anomaly (GLA; a.k.a. lymphangiomatosis) and Gorham-Stout Disease (GSD)
  17. Glucose Transporter Type 1 Defiency Syndrome (Glut 1DS)
  18. Inclusion Body Myositis (IBM)
  19. Lymphangioleiomyomatosis (LAM)
  20. Maple Syrup Urine Disorder (MSUD)
  21. Mitochondrial Complex 1 Deficiency (NUBPL)
  22. Mucolipidosis Type IV (ML4)
  23. Mucopolysaccharidoses (MPS)
  24. MPS Gene Spotlight: Mucopolysaccharidosis (MPS)
  25. Neuroendocrine Cell Hyperplasia of Infancy (NEHI)
  26. Niemann Pick Type C (NPC)
  27. Pitt Hopkins Syndrome (PTHS)
  28. RASopathies
  29. SETBP1 Disorder
  30. Snyder-Robinson Syndrome
  31. STXBP1 Encephalopathy

This is an international grant.

For any administrative inquiries regarding this grant please email Samantha Charleston.

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