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