research
28th May 2009 | 17:00
Listed below are some Adrenoleukodystrophy research notes
Max Planck Institute of Experimental Medicine – Prof. Klause–Armin Nave, Celia Kassmann, Hauke Werner in collaboration with S. Forss-Petter (Wien), J. Berger (Wien) and M. Baes (Leuven)

X-linked adrenoleukodystrophy (ALD) is the most frequent inherited CNS myelin disease (leukodystrophy) in children. This neurological disorder is caused by mutations of the X chromosome-linked adrenoleukodystrophy protein gene (Abcd1).
Severely affected ALD patients are young males with a rapidly progressing, lethal CNS demyelination but a slowly progressing non-inflammatory degeneration of spinal cord axons (Adrenomyeloneuropathy, AMN).
Both ALD and AMN patients suffer from adrenocortical insufficiency. (Addisons disease).

The responsible Abcd1 gene encodes an ABC transporter (ALDP) specific for peroxisomes. Accumulation of very long chain fatty acids (VLCFA ) is typical for this and other peroxisomal disorders, but whether this biochemical defect is the cause of the demyelination or merely a biochemical marker is not known.

Clinical Trials

Active clinical trials are currently in progress to see if proposed treatments are effective or not:
-   Glyceryl Trioleate (Lorenzo's oil)
-   Beta Interferon and Thalidomide
-   Combination of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil)
-   Hematopoietic stem cell transplantation

Lorenzo Odone

Lorenzo Michael Murphy Odone (May 29, 1978 – May 30, 2008) was probably the most famous patient with ALD.
His parents Augusto and Michaela Odone, frustrated by the limited treatment available, sparked the invention of "Lorenzo's oil", which is still being studied to see if it has therapeutic benefit in halting the destruction of the myelin sheathing of nerves caused by this disease.
PATRICK MC DONALD | katie pownall | sarah mousley | Debbie Clifford | jayne pownall | Diane Keeling | Gail Liley | Katy Robinson | johnny pownall | lee attewell | tony hoole | Sharron Lithgow | Eileen Lithgow | Emill Williams | Laila Dannourah | julie stubbings | Pauleen Johnson | jade davis | Hazel Walker | Bronwen Downing | michelle hamilton | Annabel Haigh | georgina allen | Paul Yassin | Robert Harding | laura elner | deborah mansfield | gemma woods | Megan Mansfield | sandra Mullin | sidonie emerson-quorn | stephanie stahlhut | Gill Ruane | valerie black | jamie barlow | beth attewell | Angela Alonso | Jane Bethea | Hannah Tongue | jonathan redshaw | David Bowden | Nikki Stevenson | Hannah Symmonds | James Liley | Ali Ratcliffe | ANNE MARKS | charlotte carr | Lara Newton | nicola billiald | ben radford | Lynne Powell | james pickering | nicola smith | John Dainton | Michael Sadler | JOHN GREGORY | Sophie Wale | Stephen Mason | Laila Dannourah | claire pearson | Jack Barclay | Paul Barclay | Stephanie Hemingway | joss williams | julie redshaw | Guy Gregory | alison davis | CHERRY GOODSON | Chloe Barclay | sharon griffin | regan willett | lisa radford | jean liley | Jess Robinson | Jane Manchester | jayne keeley | ellie may pownall | Ainslie sullivan | Aimi Liley | Lydia Honey | Cara Elliott | MARK PARGIN | Tony Lithgow | Rajan Gill | danielle willett | Laura Simpson | Ben Thompson | david miller | helen sullivan | Anne Walker | ian bliss | Rosie Gunn | Jo Barclay | tom willett | Thomas Goodson | Stephen Mayes | chris gregson | Nikki Carr | karen rees | chadd pearson | DANIEL PARGIN | Gwen Benintende | Sabeena Gill | Gary Billiald | JOSH PARGIN | Matthew Hannah | Angharad Bowden | GEORGIE GREGORY | amy pownall | victoria toomey | Peter Liley | Tom Mawhood | russell davis | alice redshaw | Julie Brown | ANDREW COCKAYNE | sophie green | Sam Liley | Tracy Isaac | Emma Liley | Aimi Liley | Shirley Oates | Amber Stafford | Karen Green | Jasmine Wilkinson | nicole and julia roomates | harry willett | Stuart Green | mark liley | Tracey Hamilton | Angharad Bowden | amelia green | Gillian Pear | Gillian Pear | Stacey White | Chris Sadler | francesca ritchie | Sue North | Ian Brown | Suzanne Bradley | katie addyman | DEBORAH PARGIN | steven grimmett | Sophie Oates | craig hamilton | Will Gunn | ian bliss | Sean Wiggins | Caroline Wagg | Robert Clarke | lee redshaw | helen liley | sophie probert | nicola carman | Daniel Wale | Lesley Belcher | Ben Robinson | joshua stubbing | Keith Goodson | GUY GREGORY | claudia rees | Joan Redshaw | Dave Brown | martie ronquillo | Laura Keeley | charlotte hannant | Sharon Liley | Luke Day | Abigail Parker | beth appleton | Mark Liley | SUSAN GREGORY | rachel dodds | CHERRY YASSIN | thomas radford | rebecca mcdonald | Ellis Belcher | Terry McCartney | claire Bailry | amber sullivan | Julia Barnitt | vicky greenhough-knight | Becky hempstead | Robyn Black |
£755,964.57