FDA Expands Commitment to Patients with Rare Diseases

http://www.fda.gov/Drugs/NewsEvents/ucm201994.htm

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Drug delivery breakthrough targets organelles

http://www.nanowerk.com/news/newsid=15042.php

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Another loss

It is with sadness that I report the passing of Gustav Viktor Angantyr
04/11/1994 - 02/25/2010 in Sweden. Gustav went into Hospital for
Scoliosis Surgery, and suffered heart complications. Our Warmest
thoughts and wishes extend to his family and the rest of the world wide
FA community.

iPS cells related article - NIH grant funds study on stem cells from ALS patients.

iPS cells related article - NIH grant funds study on stem cells from ALS patients.

Therapeutic Cells Derived From Reprogrammed (iPS) Stem Cells Display Early Aging

Therapeutic Cells Derived From Reprogrammed (iPS) Stem Cells Display Early Aging

social worker/project officer

http://mycareer.com.au/consumer/find/job/view.aspx?s_rid=recommendation-jobs&s=705&jobid=7371600

Social Worker/ Project Officer
Murdoch Childrens Research Institute, located at the Royal Children's
Hospital is Australia's largest child health research Institute. Our
team of 1,000 researchers is driven by the excitement of discovery and
the ambition to make a real difference to the health and well being of
children. Our world class laboratory, clinical and public health
research helps to prevent and treat conditions including allergies,
asthma, brain injury, cancer, cerebral palsy, depression, genetic
conditions, obesity, premature birth and infectious diseases.

Part-time (2 days per month)
* Flexible working hours
* Part of dynamic, multi-disciplinary team
*
The Friedreich Ataxia Clinic is a vibrant, unique multidisciplinary
clinic that provides medical and allied health ambulatory care
intervention to approximately 130 national and international children
and adults with Friedreich ataxia annually.

The successful applicant to this position will provide social work
services to the children and adults who attend the clinic. This position
is for two days per month. A proportion of these clinical hours will
comprise attendance at the Freidreich Ataxia Clinic at Monash Medical
Centre on the fourth Tuesday of the month and the remainder will involve
follow up that may be completed at a home office if desired.
Undertaking home visits to clients may be required on occasions.

A competitive remuneration package will be offered to the successful
applicants who will also be able to access the Institute's generous
salary packaging arrangements.

Applications for this Social Worker/ Project Officer position, Reference
Number MC2175 close Friday 5th March 2010.

Prof.Martin Delatycki's clarification on the FA research breakthrough at the Monash Institute

Prof.Martin Delatycki's clarification on the FA research breakthrough at the Monash Institute

Students Help FA

Hi everyone,

just letting you all know that Trinity College Gawler River School,
has, through their House - Dawkins, named Help FA in SA as their charity
for 2010!. This means that the House will spend the year, promoting and
helping and raising funds for FA!..

Way to go, Dawkins House!

Small Charity gets Big Boost

Monday 15th February.
http://fainsa.blogspot.com

Help FA in SA ( Help Friedreich Ataxia in South Australia) is delighted
to announce, AMP Foundation will once again be supporting the FA Charity
Dinner 2010.

"This means so much to us" said Laurel Hosking ( founder of Help FA in
SA). " This means we can truly make a difference in the dollars we can
give to research.... each cent gets us closer to a cure"

Friedreich Ataxia is a genetic neuro-degenerative condition that affects
about 1:30,000 people. This disease gradually robs the sufferer of
control of their legs, arms, hands, speech, swallowing, strength, and
heart function. 1 in 90 carry this devastating gene and do not know
it. Laurel and Wes Hosking, found out the hard way, they were carriers,
when their son Billy was diagnosed at age 9... Following his diagnosis,
Laurel, with the support of Wes and their four young children, started
Help FA in SA.

"I wanted to raise awareness of FA in South Australia, I hope to
connect as many FAers in South Australia as possible, and give them
information on clinics, and drug trials and research, but above all I
hope to eventually educate doctors enough that other parents do not have
to spend years searching for a diagnosis!"

All money raised goes to the Primary research body in Australia- The
Friedreich Ataxia Research Association ( Australasia) ..FARA(A).
http://www.fara.org.au

FARA and Help FA in SA is run by volunteers, and can proudly boast that
100% of money raised goes into research and actually helps finds a cure.

The Charity Dinner will be held on Saturday 14th August 2010, at the
Lakes Resort Hotel, Brebner Drive, West Lakes. Live Band :- Flaming
Sambuccas and silent auction.

Further information on the FA Charity Dinner SA 2010, or on Help FA in
SA, go to http://www.fainsa.org

--
Laurel Hosking
Help FA in SA
mob: 04204 98963
ph: 08 643 01857
laurel@fainsa.org
http://www.fainsa.org

FA Article via FARA

Hi Everyone.

More news from FARA.

" This article just came out in Neurology Now, a publication of the
American Academy of Neurologists. This publication is sent to
neurologists offices around the country (USA) and distributed in waiting
rooms and offices. The article focuses on one approach to therapy that
FARA has been supporting, HDAC inhibition. The article is not
comprehensive, it does not review all of the potential therapies,
however it does recognize the promise and progress of FA research,
specifically HDAC inhibitors, and highlights the work of Dr. Joel
Gottesfeld, a FARA funded researcher. "

http://www.curefa.org/_pdf/NeurologyNowJan2010.pdf

New Hope

Hi everyone, the following was published in The Age newspaper on
February 11th 2010. There is also video grab on this subject.

http://www.abc.net.au/catalyst/stories/2608076.htm

to read this article in context, go to
http://www.theage.com.au/national/new-hope-in-hunt-for-ataxia-cure-20100210-nsgm.html

MELBOURNE scientists have made a breakthrough in the hunt for a cure for
Friedreich's ataxia, a genetic disease with no known treatment that
causes slow nerve degeneration and early death.

Teams from the University of Melbourne and the Monash Institute of
Medical Research have made pluripotent stem cells from the skin of FA
sufferers, which could provide a shortcut to a drug-based cure or even
be used directly as a treatment.

The work has been hailed as being at the forefront of worldwide research
into a disease that affects hundreds of Australians.

Friedreich's ataxia shows up in teenage years, as the faulty gene causes
low levels of a protein vital for delivering iron to nerve and heart
cells. Sufferers gradually lose their balance and co-ordination, develop
heart problems and often diabetes.

Melbourne University's Mirella Dottori said the key to the new approach
were Monash's ''induced pluripotent'' stem cells, which had been
manufactured from the skin of the FA patients.

Dr Paul Verma, MIMR's stem cell research program leader, created
Australia's first iPS cells early last year.

Dr Dottori and her colleague Dr Alice Pebay have ''pushed'' these cells
to turn into nerve and heart cells, which can be studied to better
understand the disease.

They can also be used to test existing drugs to see if they reverse the
effect of the faulty gene. And they may even be used in ''regenerative
therapy'', by correcting the faulty gene then re-implanting the cells in
an FA patient.

''It is very exciting - new doors are opened,'' Dr Dottori said.

The research was welcomed by Carrie Beetham, 32, of Hawthorn, who found
she had FA in her teens.

''I knew I would be in a wheelchair at some stage but I didn't know it
was going to move so fast. It is about keeping yourself strong and
keeping your mind strong. The main part is trying to be positive, trying
to enjoy the life that we can enjoy, trying to remain hopeful.''

Repligen and HDAC-3

This is potentially very very exciting
This came from Pam  of FAPG

I listened to Repligen's 3rd quarter financial report this morning and
transcribed what they said about the HDAC inhibitors. Here is what was
said:

"We are also developing inhibitors of HDAC-3 for the treatment of
Friedreich's Ataxia. We have completed the evaluation of one of our
lead compounds in a 2 species toxicology study which is required by the
FDA prior to the initiation of clinical trials. Manufacturing of
clinical drug supply is in progress and we expect to file an IND to
initiate Phase I clinical trials by mid-2010. These studies are being
partially supported by the MDA which provided us with a second research
grant in December. Our clinical goal is to cross over from normal
volunteer studies to Friedreich's patients as soon as possible to enable
us to get an early read on the potential efficacy of our drug by
monitoring changes in the blood levels of frataxin, the protein missing
in Friedreich's patients. Recent data suggests that this biomarker is
expressed at a consistent level in patients which will facilitate the
observation of a potential increase in frataxin levels following drug
treatment. We believe that there are 15,000 Friedreich's Ataxia
patients worldwide which represents a market opportunity of more than
$300,000,000 even with very modest pricing assumptions."

This is very exciting news!
Pam

Team FARA

An message from FARA

Team FARA Races Across America to Raise Awareness for Friedreich's
ataxia

February 4, 2010- The Friedreich's Ataxia Research Alliance is fielding
a team of four cyclists in the "world's toughest bicycle race" - Race
Across America (RAAM). Beginning on June 12, 2010 in Oceanside, CA, Team
FARA will ride in a relay format until reaching the finish in Annapolis,
Maryland approximately 9 days later. Over 215 RAAM competitors will
travel a 3,000+ mile route with 100,000 vertical feet of climbing at an
average minimum speed of 14 mph.

Team FARA in RAAM consists of 4 members- FARA's Spokesperson and Ride
Ataxia Program Director- Kyle Bryant as well as Ride Ataxia cyclists-
Sean Baumstark, John Lockwood and Mike Mellott. Bryant states,
"Sometimes it takes a crazy idea to get noticed. Our Team is excited to
use our RAAM journey as a vehicle to draw attention to the rare disease
which affects both me and Sean and so many others. We also hope that it
encourages all people- with and without physical limitations to maintain
a healthy and active lifestyle and challenge their preconceived notions
of what is possible."

Team FARA's strategy for crossing the country in under 9 days is to
create 2 man sub-teams that ride for 8 hour shifts, alternating 30
minutes each on the road. This will allow the team to have at least one
rider on the move 24 hours a day. This endeavor will require a great
deal of planning and coordination from the team's 8 person support crew
as well as the assistance of the FA community.

Team FARA in RAAM needs your help. YOU can support the team with:

Sponsorships
* Volunteering
* In Kind Donations
* Local Media Outreach
* Support Along the Route
*
To find out more about the team, the route they will travel, and how you
can help please visit http://teamfara.blogspot.com

About RAAM
RAAM is currently the most recognized, longest endurance cycling event
in the world, and garners worldwide respect for the sheer magnitude of
its fitness challenge. www.raceacrossamerica.org

About Ride Ataxia
The mission of Ride Ataxia is to Educate the public about ataxia by
drawing attention through acts of physical endurance, Enable the
advancement of ataxia research through collaborative financial support,
and Empower ataxians by inspiring, motivating, and providing
opportunities to develop physical and mental strength.

There will be four Ride Ataxia events in 2010- Tampa Bay, FL, April 13;
Northern California- Folsom, CA, May 15-16; Philadelphia, PA October
TBD; Southern California- Los Angeles, CA, December 3-4.
www.rideataxia.org

About FARA
The Friedreich's Ataxia Research Alliance's (FARA) mission is to marshal
and focus the resources and relationships needed to cure FA by raising
funds for research, promoting public awareness, and aligning scientists,
patients, clinicians, government agencies, pharmaceutical companies and
other organizations dedicated to curing FA and related diseases.
www.CureFA.org

Contact
Jennifer Farmer
Executive Director, Friedreich's Ataxia Research Alliance
(484) 875 3015
info@curefa.org

Go The TAN

HI everyone,

just a quick reminder about Go The Tan, on the 12th and 13th
February.... Walk, Run, Crawl, Ride your way around this picturesque
Running track in the beautiful City of Melbourne. All Money raised
helps to find a cure for FA.... so take a moment out of your day, and
get some fresh air, and make a difference!
http://www.starttofinish.com.au/default.aspx?s=eventdisplay&id=1102

--
Laurel Hosking
Help FA in SA
mob: 04204 98963
ph: 08 643 01857
laurel@fainsa.org
http://www.fainsa.org

New FARA Logo (US)

Hi everyone

here is some news from FARA (US)

Introducing the new FARA logo

Dear FARA community, the FARA Staff and Board is pleased to announce the
introduction of an updated FARA logo. This mark replaces the previous
logo for all FARA printed and web materials. It is our hope that you
find this new mark to be distinctive, inclusive and identifiable.

Why a new logo?

Over the past year, FARA received feedback that our logo needed to be
updated. We were fortunate to have access to the advice of advertising,
marketing, graphic design and fundraising professionals. After getting
to know FARA , they all recommended an update to the logo, in order to
more accurately convey the culture and mission of our organization. This
became even more necessary as FARA is working with a growing number of
academic, pharmaceutical, government and advocacy organizations, and
expanding our corporate partnerships. Also, many of our fundraising
efforts have their own logo or symbol and we wanted a mark that
supported this but also clearly identified our organization.

The Process

Members of the FARA Board and Communications committee formed a team to
assess the current logo's attributes and to develop the goals for the
new logo which follow:

Present an image of a sophisticated, capable, international
research organization
* Create a clean and professional image that is easy to recognize
and reproduce
* Make the full logo readable at all sizes
* Retain some elements of the original FARA identity
* The logo should be easily compatible with other event logos
*
One aspect that you may notice is that there is no additional graphic
symbol or mark such as the children in our previous logo. While we all
miss the children and the powerful story they represent, the decision
was made to go with a simpler logo so that the FARA logo would be easily
compatible with the dozens of event logos used around the country.

The Roll-Out

As you will note, the FARA logo is already starting to appear. FARA will
gradually be changing out all communications materials. Out of respect
for our environment and hard-earned funds, we will not be throwing away
any printed materials!

All fundraisers, event coordinators and partners will receive a resource
kit for use of the logo. It will contain the new logos in electronic
formats for use in printing professionally and at home in color and
black and white.

If you have any questions, please do not hesitate to contact us. This
project was intended to enhance all of our communications efforts.

Thanks in advance for your assistance!

About FARA
The Friedreich's Ataxia Research Alliance's (FARA) mission is to marshal
and focus the resources and relationships needed to cure FA by raising
funds for research, promoting public awareness, and aligning scientists,
patients, clinicians, government agencies, pharmaceutical companies and
other organizations dedicated to curing FA and related diseases.
www.CureFA.org

Contact
Jennifer Farmer
Executive Director, Friedreich's Ataxia Research Alliance
(484) 875 3015
info@curefa.org