Founded by Sherine and Mark Blackford in honor of their 3 year old son Loic, the Loving Loic Foundation seeks to develop a gene therapy for pantothenate kinase-associated neurodegeneration (PKAN).

PKAN can present in two ways, classic and atypical. Classic PKAN is characterized by early-childhood onset of progressive dystonia, parkinsonism, and brain iron accumulation with children ... more

Founded by Sherine and Mark Blackford in honor of their 3 year old son Loic, the Loving Loic Foundation seeks to develop a gene therapy for pantothenate kinase-associated neurodegeneration (PKAN).

PKAN can present in two ways, classic and atypical. Classic PKAN is characterized by early-childhood onset of progressive dystonia, parkinsonism, and brain iron accumulation with children bound to a wheelchair and placed on a feeding tube at the end of life. It is terminal and children with classic PKAN typically do not survive past age 10. Atypical PKAN has a later onset, often around adolescence. Atypical PKAN has a more gradual progression and children have longer life expectancies.

With the help of the Loving Loic Foundation, a gene therapy has been developed and $1.4 million has been raised. Partnering with Oregon Health & Science University and UMass Chan Medical School’s Horae Gene Therapy Center, the Foundation leverages the expertise of experienced scientists to develop an effective treatment for PKAN. The immediate goal is to raise an additional $3.6 million get the cure to human clinical trials. Developing an effective gene therapy will not only cure PKAN, but opens the donor further to developing gene therapies for the over 30 million Americans affected by rare diseases.

Learn more at lovingloic.org.
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