Eliza Fights Niemann Pick Type C in Poland, A Fight Her Brother Lost at Age 14

Niemann_Pick_C_Eliza

Eliza is 12 years old and suffers from Niemann-Pick disease, type C. It is an inherited disorder which attacks the brain and leads to gradual loss of all biological functions. A rare inherited disorder, with only a couple 100 ill children in Poland and worldwide, [...]

Update: Orphan Drug Application (hydroxy propel beta cyclodextrin) by Chris Hempel

From Chris Hempel (Addi & Cassi Fund). Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. [...]

Niemann-Pick Disease: An Update on Joseph Colton

Just Testing the Captions

Our 4 year old son Joseph Colton has Niemann-Pick disease type A/B.   The Niemann-Pick (NP) diseases belong to a family of 40+ inherited disorders identified as lysosomal storage diseases or LSDs.  There are two distinct sub-families of Niemann Pick diseases.  NPA and NPB diseases are [...]