FA Hope abounds

Hi Everyone,

I wanted to give you some general feedback into what was discussed at
the FARA Review Meeting last Thursday.

The Researchers and Scientists covet their studies very closely, so
anything I say here will be of a general nature out of respect for their
work.

Stem cells were the major theme for this years meeting. With many
interesting developments, including research into avoiding rejection of
stem cells by using one owns stem cells,and removing the affected FA
part.

Other Stem Cell work uses Virus to help with the reproduction of cells.
Apparently the virus stops dead after initiating cell growth.

EpiGenetics is fascinating. Instead of using regular gene therapy, it
uses an inhibiting agent to attach to the gene and turn the gene off. A
compound to turn the FA gene off, was found in UCLA last year... so that
is very exciting.

lab work is continuing on the Frataxin protein with promising findings
on Frataxin levels in FA patients, Carriers and non carriers.

Trials abound. Word on Idebenone ( for those not already using it) is
that it should be available to patients by the end of 2009.
Deferiprone trial is about to start in australia. Unfortuately there
are only 2 definate participants with 2 more maybes. they had hoped for
10, but will add this data to the worlds. If you know anyone between
7-17 with FA they may be interested in participating.

Other trials are happening around the world with some held up in
Ethics...

Antioxidants... some research is suggesting that antioxidants are not
the panacea we had hoped, however, idebenone DOES target the heart in a
positive way, and help some neurological aspects so there is a
suggestion there are factors at work other than straight antioxidants.

FA testing models were discussed with the conclusion that "the wheel" is
still not perfect and still upto fine tuning.

Audiology. WOW:- there was a presentation on this front which was
eyeopening. It has been found ( for the most part) that FA sufferers
have normal hearing on general sound testing... even cochlear testing
brings normal results.

However, when the time delay of hearing was tested, it was found that FA
sufferers often had problems with the speed they " hear" the sounds...
this then leads to the speed of processing the sound, and it was found
in general, that FA sufferers depending on their hearing speed had
problems with processing what they hear.... for eg, the more background
noise there is, the harder it is to distinguish certain aspects of
speech.. What is not being tested generally is this hearing speed/
processing speed..... So if you attend the clinic, it may be worth
asking Louise ahead of time if you can get this tested.

On the clinic, they have about 120 attending the clinic, with most from
Vic, NSW and QLD, and 4 from SA ( its a start)... none from WA as yet,
but distance is a considerably factor. On that front... you do all
realise that you can apply for distance rebate on your travelling and
accommodation to attend the clinic. Just ask Louise Corben about it.

Unfortunately, as the day was running a bit overtime, I had to leave to
catch a plane, and missed most of Louise Kenetic presentation, but as
you can see above there is HOPE, and they ARE getting closer.... the
fact that Idebenone is so close to being available and that we now have
a trial happening in Australia is a huge step.

Hope this has provided you with something interesting... if you have
further questions I will try and shed some light on them... still
processing it all myself.

Merry Christmas to you all, and hope and happiness for 2009

--
Laurel Hosking
Help FA in SA
mob: 04204 98963
ph: 08 85221167
fax: 08 85221313
laurel@linvid.com

Drug Trial here in Australia

Well it is finally here!.. Recruitment is starting for a "six-month
double-blind, randomized, placebo-controlled study investigating the
safety and tolerability of deferiprone in patients with Friedreich's
ataxia. "

This is a world wide study being run in Europe, Australia and Canada.

Criteria seems to be "7 to 35 years of age, has a confirmed genetic
diagnosis of FRDA with number of GAA repeats greater or equal to 400 on
both copies of DNA, has a score between 20 and 85 on a neurological exam
known as Friedreich's Ataxia Rating Scale (FARS), has a negative urine
pregnancy test (if applicable) before the research study starts and is
currently not participating in another research study. "

further information, on

Name: Ms Geneieve Tai

Role: Research Coordinator

Telephone: (03) 8341 6374

cheers all

--
Laurel Hosking
Help FA in SA
mob: 04204 98963
ph: 08 85221167
fax: 08 85221313
laurel@linvid.com

News since the ball...

Hi,

Next month I will be attending the Grants round meeting of FARA(A) in
Melbourne. I recently had a wonderful luncheon meeting with the
Executive Director Varlli Beetham. She had come across to SA to speak
to Trinity College students, who have embraced FARA as their yearly
fundraiser. During this Assembly, the students presented Ms Beetham
with an initial chq for almost $400.... not bad for a couple of gold
coins thrown in a classroom jar.

As a part of this presentation, Billys slide show on FA was shown to the
entire school, and now the major fundraiser of Tea towels is about to
kick off.

As part of our meeting, we discussed the importance to get neurologists
on side here in SA. This is vital for the establishment of any drug
trials here in SA, plus the long term goal of an SA clinic.

As you can guess, I " dobbed in" billys neurologist, who has accepted an
invitation to attend the grants meeting and meet with FARA and be
involved first hand.

As you can see, things are still ticking along.

cheers everyone

Laurel

next years event

Hi everyone.

a bit of advance notice... next years fundraising event is to be held on Saturday 1st August from 7pm... more details to follow closer.

cheers all
Laurel

Ball Summary

Hi everyone,
I wanted to share the success we had at our first Ball for FARA, here in
South Australia.

It was an smallish affair, but BOY did we make up for it with laughter
and frivolity.
The evening started with the MC, demanding a $10 donation everytime
someones mobile phone rang, and continued in this VERY merry way to
people throwing in $50 notes for a raffle for their table centre ( vase
of flowers).
The Silent Auction saw bidding wars, which almost lead to " marker pens
at 10 paces", and the Band, encouraged two men from the audience to come
up and be the " boys from ABBA"... need I say more?

At midnight the band finnished up, much to the dismay of the guests, and
the party moved into the neighbouring Bar for the next few hours.

Big Thankyou to A/Prof. Martin Delatycki for attending and speaking.....
He opened alot of peoples eyes, who then opened their wallets.

The best thing, is that everyone wants a repeat next year.

Cheers everyone

Laurel

news!

http://www.santhera.com/index.php?docid=212&vid=&lang=en&newsdate=200807&newsid=1238090&newslang=en

Published: 07:00 24.07.2008 GMT+2 /HUGIN /Source: Santhera
Pharmaceuticals Holding AG /SWX: SANN /ISIN: CH0027148649

July 24, 2008: Santhera Receives First Product Approval: Health Canada
Approves Catena® for Treatment of Friedreich's Ataxia

Santhera Pharmaceuticals (SWX:SANN), a Swiss specialty pharmaceutical
company focused on neuromuscular diseases, announced today that Health
Canada has approved with conditions SNT-MC17/idebenone for the treatment
of Friedreich's Ataxia. Health Canada's decision is the first marketing
authorization worldwide for any Friedreich's Ataxia therapy. The new
drug will be marketed in Canada under the brand name Catena®. Santhera
has established a wholly owned Canadian subsidiary, Santhera
Pharmaceuticals (Canada), Inc. recently incorporated in Montréal,
Quebec, to address the needs of Canada's several hundred patients with
this rare and severely progressive muscle disease. Launch of Catena is
anticipated for the end of October 2008.

In clinical studies submitted to Health Canada as part of the approval
process, Catena showed statistically and clinically relevant
improvements in Friedreich's Ataxia patients, as measured by Activities
of Daily Living scores as well as cardiac and neurological functions.
The approved product labeling allows for the treatment of symptoms of
Friedreich's Ataxia. Two doses are approved: a starting dose of
450 mg/day for patients below 45 kg body weight and 900 mg/day for
patients above 45 kg body weight whereby treating physicians have an
option to dose up to 1,350 mg/day for patients below 45 kg body weight
and up to 2,250 mg/day for patients of more than 45 kg body weight if
needed. Under the conditions of the Notice of Compliance with
Conditions, Santhera has agreed to submit additional data from its
ongoing phase III clinical trial in the United States to confirm the
efficacy of the therapy.

Klaus Schollmeier, Chief Executive Officer of Santhera said: "We are
very pleased about this market authorization by the Canadian authority.
Health Canada concluded that the data presented were promising enough to
allow Catena's approval for the benefit of patients now while requiring
confirmatory efficacy data when they become available. Today's approval
is a major milestone for the Friedreich's Ataxia community in Canada and
elsewhere. For the first time, physicians will be able to offer patients
an approved, safe and efficacious therapy to treat their devastating
disease." He continued: "This first marketing authorization marks a
significant event for our Company, one that has been our goal since the
inception of Santhera in 2004. Today, Santhera's vision of offering
therapies for orphan indications is becoming reality. The entire team at
Santhera is energized by this success and is even further encouraged to
work towards approvals in other regions."

MJ Roach, VP Marketing & Sales and Santhera's General Manager for North
America, commented: "Canada's marketing authorization provides an
excellent foundation to establish a medical and marketing platform in
North America for the treatment of rare neuromuscular diseases in
general and for Friedreich's Ataxia in particular. The Canadian market
will also provide valuable insights for launching the product in the
United States, once the ongoing clinical trial is completed and the
product is approved. We look forward to working with physicians in
Canada who prescribe Catena to Friedreich's Ataxia patients."

Update on ongoing phase III clinical trial

In the United States, the IONIA (Idebenone effects On Neurological ICARS
Assessments) phase III trial has currently enrolled 41 patients. It was
agreed with the US Food and Drug Administration under a Special Protocol
Assessment process to recruit a minimum of 51 patients but to include
more patients if available. Given the current prospects for patient
availability, Santhera and its US clinical investigators believe that
the final study will include about 60 to 65 patients.

Conference call

At 19.00 CET / 18.00 UKT / 13.00 EST on July 24, 2008, Santhera will
host a conference call. Anyone interested in participating may join the
teleconference facility using the following dial-in in Switzerland
+41 52 267 07 36. The conference call will be recorded for playback and
is available one hour after the conference call ends and for 20 days
under +41 52 267 07 00 (reference no. 668713).

About Friedreich's Ataxia

Friedreich's Ataxia is a rare but severe genetic neuromuscular disorder
that results in the degeneration of an individual's nerve and muscle
tissue. This disorder causes loss of muscle control, uncoordinated
movements, muscle wasting and thickening of heart walls which frequently
leads to a shortened life span. Friedreich's Ataxia affects both
Caucasian males and females equally and it is estimated that about
20,000 patients suffer from the disease in both North America and
Europe. Average life expectancy for Friedreich's Ataxia patients is
limited to approximately 35 to 50 years.

The disorder results from a genetic defect in the gene encoding for
frataxin. Reduced levels of this protein ultimately result in impaired
energy production in mitochondria, the cells' energy production centers,
and elevated oxidative stress. Tissues that have the highest need for
energy, in particular nerve and cardiac tissues, are primarily affected
by frataxin deficiency resulting in pathological changes in heart muscle
anatomy and function and loss of nerve cells.

About Catena®

Catena may be useful in the symptomatic management of patients with
Friedreich's Ataxia. The drug is believed to increase the supply of
energy to cells in the body. Additionally it has antioxidant properties
and may protect the cells in the body which are damaged by the disease.

Catena® is a trademark of Santhera Pharmaceuticals, registered in Canada
and the United States.

About Notice of Compliance with Conditions

A Notice of Compliance with Conditions (NOC/c) is an authorization to
market a drug in Canada issued by Health Canada, indicating that the
sponsor has agreed to undertake additional studies to confirm the
clinical benefit of the product. A market authorization under the NOC/c
policy allows Health Canada to provide earlier market access to
potentially life-saving drugs. Eligibility for an NOC/c is restricted to
promising new drug therapies intended for the treatment, prevention or
diagnosis of serious, life-threatening or severely debilitating diseases
or conditions for which a) there is no alternative therapy available on
the Canadian market or, b) where the new product represents a
significant improvement in the benefit/risk profile over existing
products. Once a sponsor provides satisfactory evidence of the drug's
clinical effectiveness, and all the conditions agreed upon have been
met, Health Canada will remove the conditions associated with market
authorization in favor of a full approval.

* * *

About Santhera

Santhera Pharmaceuticals (SWX:SANN) is a Swiss specialty pharmaceutical
company focused on the discovery, development and marketing of
small-molecule pharmaceutical products for the treatment of severe
neuromuscular diseases, an area of high unmet medical need which
includes many orphan indications with no current therapy. Santhera
currently investigates three compounds in five clinical-stage
development programs. The Company's first product, SNT-MC17 (INN:
idebenone) has received a marketing approval with conditions from Health
Canada to treat Friedreich's Ataxia and will be marketed under its brand
name Catena. The product is also under review by health authorities in
the EU and in Switzerland for the same indication, while in the United
States a pivotal phase III trial is recruiting patients.
SNT-MC17/idebenone has also shown efficacy in a phase II clinical trial
as a potential treatment for the indication Duchenne Muscular Dystrophy.
For further information, please visit www.santhera.com.

For further information, contact

Klaus Schollmeier, Chief Executive Officer

Phone: +41 (0)61 906 89 52

klaus.schollmeier@santhera.com

Barbara Heller, Chief Financial Officer

Phone: +41 (0)61 906 89 54

barbara.heller@santhera.com

Thomas Staffelbach, Head Public & Investor Relations

Phone: +41 (0)61 906 89 47

thomas.staffelbach@santhera.com

Disclaimer/Forward-looking statements

This communication does not constitute an offer or invitation to
subscribe for or purchase any securities of Santhera Pharmaceuticals
Holding AG. This publication may contain certain forward-looking
statements concerning the company and its business. Such statements
involve certain risks, uncertainties and other factors which could cause
the actual results, financial condition, performance or achievements of
the company to be materially different from those expressed or implied
by such statements. Readers should therefore not place undue reliance on
these statements, particularly not in connection with any contract or
investment decision. The company disclaims any obligation to update
these forward-looking statements.

News release Approval Canada

more on A0001

Below are two emails... one from Paul Konanz and the other from US FARA
president Ronald Bartek
-----------------------------------------------------

Hello all,

I'd like to follow Paul's excellent comments with some information FARA
has obtained about the A0001 trial. This information reinforces Paul's
points about the format of this trial as compared with the Idebenone
trial and may help answer some of your questions.

The phase I trial of A0001, as stated in the release, is in healthy
volunteers. It is being conducted by a Contract Research Organization
(CRO) that was chosen by Penwest Pharmaceuticals and with which FARA has
no dealings. This is, by the way, the more typical way phase I trials
are conducted (by CROs in healthy volunteers that they have on their
rolls). I believe phase I of the Idebenone trial was conducted,
atypically, in FA patients primarily because Idebenone had already been
"in a lot of people." Idebenone had been tried at low doses in a number
of other diseases such as Parkinson's, Huntington's and Alzheimer's, and
had been taken by a lot of healthy people as an anti-aging or
brain-function aid. So, the FDA agreed the Idebenone phase I could be
in FA patients. Similarly, the iron chelator Deferiprone and EPO have
both been "in a lot of people" with other indications, so the phase I
work, or pilot studies, in those two drugs have been conducted in FA
patients.

A0001, however, has not been "in a lot of people" so the FDA suggested
beginning with the typical phase I approach - in healthy
volunteers. FARA has not seen the protocol for this phase I study of
A0001 and would not usually need to see it. My understanding is,
though, that carrier status would usually disqualify a potential
participant. I believe healthy participants are usually young students
with no involvement at all with the disease in question. CROs normally
recruit the healthy volunteers very quickly from their own rolls and ads
and have no trouble doing so. This A0001 phase I trial, for example, is
to use only 60 healthy volunteers and 10 of them were dosed on the very
first day (last Friday).

As Paul states, phase I of this trial, like all phase I trials, will
focus on safety. Like the phase I of Idebenone, it will look at safety
in a dose-escalating manner and will attempt to identify the maximum
tolerated dose. Data from this phase I trial will be used to establish
safety of A0001 in humans and will help instruct selection of the best
doses to use in the phase II in FA patients.

We will ALL be VERY much involved in the phase II of the trial, of
course, and FARA will let everybody know about timing and
inclusion/exclusion criteria, etc. We are in constant contact with the
drug companies involved, on a daily basis, and will be working very hard
to accelerate their timelines. FARA's Scientific Advisors and Board of
Directors meet at the end of next week and we will review, based on
reports we are now receiving from all the drug companies, the schedules
and timelines for all the clinical trials currently underway and coming
up. Of course, we will let everybody know the results and how you can
help.

In preparation for all these clinical trials, please make sure all FA
patients are signed up in the FARA Patient Registry and for FARA's
electronic bulletins and newsletters.

Hope this helps.

Warm regards to all,

Ron

(Ron & Raychel; Keith-22-FA; Byron-24-clear; Stuart-19-carrier)

Ronald J. Bartek
President
Friedreich's Ataxia Research Alliance (FARA)
P. O. Box 1537
Springfield, VA 22151
Tel (703) 426-1576
FARA website: http://www.CureFA.org

Email: fara@CureFA.org

Please register in the FARA Patient Registry at
http://www.curefa.org/registry/

and for e-news at
http://visitor.constantcontact.com/email.jsp?m=1101190303489

--- On Wed, 7/16/08, Paul Konanz wrote:

From: Paul Konanz

Subject: [FAPG] Edison A-0001 Phase I Trial Participants
To: fapg@fortnet.org, internaf@yahoogroups.com
Date: Wednesday, July 16, 2008, 10:11 PM

Reminder on the Edison A-0001Phase I Trial

It is wonderful news that one more possible FA treatment is starting FDA trials, with the announcement of the Edison A-0001 Phase I trial.
One note is that this Phase I trial is to completely focus on safety in human subjects using non-FA-involved participants. In the press release
it says, "The phase I clinical trial just initiated by Penwest Pharmaceuticals will be conducted in healthy volunteers...".

For sure this means no FA'ers will be involved and very probably no carriers (parents, siblings, etc) either. I have little information
on the protocol at this time (this trial is not on the NIH trial page). Looking at the Idebenone Phase I trial as an example can give you a feel
for what is involved in a safety-testing-only trial. That trial was split into an A and B sections, with A being a single dose to volunteers with
close scrutiny for some time after the dose, and B was several doses up to 75mg/Kg/day for one month with the same close scrutiny.
DO NOTE FOR IDEBENONE THEY USED FAers.
They aren't with the A-0001, according to the press release. If you are interested, the Idebenone Phase IB trial description can be found here,
http://www.clinicaltrials.gov/ct2/show/NCT00078481?term=Friedreich%27s+Ataxia&rank=4

So, please don't try to volunteer for this A-0001 trial if you think you have one or more bad FA-related genes. Several of the FA
organizations have received many inquiries but have little information because they are not involved and we don't qualify anyway. :-)

These organizations did comment how they appreciated the interest and willingness of the families to so quickly contact them!
It demonstrated how closely tied in to advances in research we are.

Regards,

Paul