FDA Grants Clearance for Experimental Intrathecal Administration of Cyclodextrin

The FDA has granted clearance of an Investigational New Drug (IND) application for administration of Hydroxypropyl Beta Cyclodextrin (cyclodextrin) into the central nervous systems of two patients with Niemann-Pick Disease Type C.

The cyclodextrin will be given intrathecally (into the space under the arachnoid membrane of the brain or spinal cord), first via lumbar injection, and then into the brain’s ventricle system. Children’s Hospital & Research Center of Oakland, California, issued a press release, which can be accessed from the NNPDF's NewsLine page.

The NNPDF would like to note the following regarding cyclodextrin:

1. Cyclodextrin has shown promise as a potential therapeutic in animal trials. However, issues of toxicity have also arisen.
2. The NNPDF is aware that cyclodextrin is being used on a single-patient Investigational New Drug (IND) basis in the U.S. and in Brazil.
3. More research is needed to understand issues related to drug delivery, efficacy and safety.
4. The NNPDF will keep the NPC community apprised of developments regarding cyclodextrin.

Amy Dockser Marcus published this related article in her Wall Street Journal Health Blog on September 23.

For more information about cyclodextrin, cyclodextrin's orphan drug designation, a glossary of terms, and links to the FDA, please visit our cyclodextrin page.

Daftar Adsense Murah

Daftar Adsense Murah Rp.50.000,-

Aktris Ini mirip sekali dengan Luna Maya

Sabai Morschek (Foto:Dok.Cariartis.com) 

 

Wajah cantik Sabai Morschek sekilas mirip Luna Maya. Disebut bermuka mirip kekasih Ariel itu, Sabai enggan aji mumpung.

"Aku mah enggak mau ambil untung. Aku juga enggak mau aji mumpung. Terserah orang-orang mau bilang apa," ujar Sabai yang dijumpai di Planet Hollywood, Jakarta, Kamis (23/9/2010).

Aktris yang memerankan wanita pekerja seks komersil di film Sang Dewi ini tak mau ambil pusing dengan pendapat orang akan dirinya.

"Aku sih cuek aja. Jalani aja semuanya masing-masing. Orang mau bilang apa, terserah. Yang penting aku mau menjalani semuanya dengan pribadi sendiri," tegasnya.

Perempuan kelahiran Padang, 3 Oktober 1988 ini baru saja membintangi film terbaru berjudul Sweetheart. Berperan sebagai Imel, karakter antagonis yang jahat, Sabai merasa tertantang.

"Aku memang merasa tantangannya gede banget di film ini. Aku enggak perlu observasi soalnya karakternya tantangan banget buat aku. Paling aku tanya-tanya saja untuk mendalami peran," ucap lulusan Universitas Pelita Harapan ini

source: okezone.com

Lindsay Lohan di penjara selama 15 jam

Aktris cantik ini di bebaskan karena gagal dalam pembuktian kasus narkoba pada hari jumat kemarin (24/9)

Lohan dibebaskan dari Rumah Tahanan Regional di pinggiran Los Angeles, Lynwood, beberapa waktu menjelang tengah malam, seperti dilaporkan jejaring selebriti TMZ.

Lohan, 24 tahun, dengan tangan diborgol digiring dari ruang sidang Beverly Hills pada Jumat pagi karena hakim menemukan bukti penyalahgunaan zat terlarang oleh bintang The Mean Star ini. Bukti penyalahgunaan obat-obatan terlarang dengan ancaman penjara tiga tahun itu adalah yang ketiga kalinya dilakukan Lohan dalam tiga tahun belakangan ini.

Hakim memerintahkan Lohan dijebloskan ke penjara hingga pemeriksaan pada 22 Oktober. Namun belakangan putusan hakim itu diubah oleh seorang penyelia dan Lohan harus membayar uang jaminan sekitar Rp 2,7 miliar.

Selama akhir Juli dan Agustus, Lohan telah menjalani dua pekan dari 90 hari hukuman penjara, dan kemudian 22 hari program rawat-inap di pusat rehabilitasi narkoba.

Seperti Inilah Kerangka Manusia Prasejarah Di Aceh

 

Sebuah peneliti telah menemukan  kerangka manusia prasejarah yang amat langka,Balai Arkeologi (BALAR) Medan, Sumatera Utara, mengemukakan kawasan Kampung Mendale, Kecamatan Kebayakan, Aceh Tengah, pernah dihuni manusia zaman prasejarah. Pernyataan ini diungkapkan setelah para peneliti itu menemukan batu kapak persegi dan sejumlah tulang manusia di ceruk (gua) hulu Danau Lauttawar. 

Dua peneliti senior dari BALAR Medan, Ketut Wiradnyana dan Lucas Partanda Koestoro, di Takengon Minggu (26/9) mengatakan, banyak ceruk di Mendale yang kini sedang digali (eksavasi) ditemukan beberapa kepingan budaya masa lalu, seperti tulang, gerabah, molusca bahkan tulang manusia dan kerangka.

"Salah satu temuan terbaru adalah kerangka manusia di Ujung Karang Kebayakan yang merupakan titik penggalian baru setelah ceruk Mendale," kata Ketut. Dalam penelitian ini ia mengajak bersama enam arkeolog lainnya.

Menurut Ketut, temuan kerangka manusia di Ujung Karang dalam kondisi terlipat belum dapat dipastikan umurnya karena masih dalam proses penggalian.

"Mungkin hari Selasa mendatang semua bagian kerangka manusia tersebut akan dapat dilihat secara utuh," kata Ketut.

Kata dia, dengan temuan kerangka manusia di Ujung Karang Kebayakan berarti sudah dua kerangka manusia ditemukan BALAR Medan di dua lokasi berbeda, Ceruk Mendale dan Ceruk Ujung Karang.

"Semakin menarik temuan di Aceh Tengah. Semoga dapat membuka tabir sejarah masa lalu dari temuan sisa-sisa atau sampah masa lalu," sebut Lucas.

Rasa penasaran peneliti arkeolog Sumatera dari BALAR Medan semakin besar setelah salah seorang warga Takengon Zulkifli MD, mengatakan kepada peneliti dari BALAR bahwa di daerah Umang Isaq Kecamatan Linge ditemukan beberapa bentuk tulisan (goresan) di atas batu.

"Saya yakin tulisan tersebut bukan dibuat sekarang karena batunya sudah berlumut dan bentuk tulisan/goresan yang tidak saya pahami," kata Zulkifli.

Menurut para peneliti arkelolog, guna mengetahui umur, semua temuan di seputaran Mendale, diperlukan analisa karbon di Badan Tenaga Atom Nasional (BATAN).

source: tempo.interaktif.com

Cassandra Phillipa Stallard

Cassandra Phillipa Stallard (NPC)
May 30, 1989 - September 21, 2010



We are very sad to learn of the death of 21-year-old Cassie Stallard due to the effects of Niemann-Pick Disease Type C (NPC).

According to Cassie's mom, Trish, Cassie died peacefully in her sleep after a day enjoyed strolling on the beach and a trip to a favorite coffee house.

All our sympathy to the Stallards and their extended family and friends in their loss.

To learn more about Niemann-Pick Disease, please visit the National Niemann-Pick Disease Foundation Web site.

Online Auction Site LIVE! Your help is requested!

The NNPDF's Niemann-Pick Disease Awareness Online Auction site is now live and operational!

Please visit the NNPDF October Awareness Month Online Auction site now and see what we are cooking up for October 1st, when opening bids will be accepted.

New auction items are being added daily, and you can begin window shopping today! Some exciting items already posted include an autographed Brett Favre Minnesota Vikings jersey, a handmade quilt, authentic Chinese wall scrolls and masks, a DVD of the Super Bowl Champion New Orleans Saints, and gift cards from R.E.I. and Target.

The list is growing daily as families around the U.S. and Canada are busy making, collecting and donating items for this important event which will raise funds in support of family services programs and essential research into Niemann-Pick Disease.

Please Help!
Your participation is needed to help make this auction the huge success we know it can be!

Please think about what you and your family might donate to the auction. Popular auction items include handmade crafts and gifts, gift cards to nationwide retailers or service providers (restaurants, department stores, home improvement stores, hotels, bookstores, carpet cleaning, etc.), sports memorabilia, collectors’ items, vacation packages, tickets to sporting or theater events, jewelry, artwork, tools, etc.

Items may be donated by an individual/family, or donations may be solicited from your area businesses.

(Please, no glass or fragile items that may be damaged in shipping, no alcohol or firearms, and nothing that cannot be shipped or for which shipping costs would be prohibitive.) (Shipping costs will be the responsibility of the buyer, unless the donor elects to donate shipping costs.)

Auction to Feature State-Themed Baskets
One special feature of the NNPDF auction will be state-themed “baskets,” or “collections,” with items unique to a state or region. Examples might be non-perishable food items representative of a state or region, regional-flavor clothing or jewelry, professional or college sports memorabilia or tickets, souvenir/collectors’ items, etc.

How to Donate
Please let us know ASAP what you plan to donate for the auction, so we can post it to the site. Please complete the form available on the NNPDF Web site and send the form, together with a photo (jpg format if possible), to NNPDF Board Member/Development Committee Co-Chair Ryan Kelly.

Please do NOT send auction items to the NNPDF; items will be shipped directly from the donor to the winning bidder at the close of the auction (October 31st), with the buyer responsible for the cost of shipping. (Donors may choose to cover the cost of shipping, particularly for small or lightweight items which cost little to ship.)

Spread the Word!
Please email the link to the NNPDF October Awareness Online Auction Site to all your friends, family and acquaintances so they can get in on the fun and excitement, as well! This is a fabulous opportunity to raise significant funds for the Quest for a Cure, but we need your help in getting the word out.

Contact Us
Please feel free to contact the NNPDF Central Office with any questions you may have about the Online Auction. We look forward to working together with you on this exciting opportunity! Together, we can make a difference!

Home from vacation

We had a wonderful time in Florida!!! It was truly a vacation and a great experience.

We enjoyed Disney World with Aaron and Andrea and their family and went to most of the

attractions like Hall of Presidents, Buzz Lightyear's Space Ranger..Haunted

Mansion...Hollywood Studios...Animal Kingdom...and the EpCot Center. We got pictures

of the Beatles singing in the park and also in France we saw the Sein River. We

really enjoyed eating at the Garden Restaurant where the Disney characters came around

to the tables and gave us one on one attention. The kids loved it. The food was

healthy and super good. Aaron and Andrea really spoiled us.

We also got to visit Elder Ryan Merkley who is serving in Orlando and I gave him a big

hug and embarrassed him probably. He looks good. Also, we got to visit Richard and

Nancy Randall on their mission at the Deseret Ranch. They made a healthy and yummy

lunch for us (lentils, fruit salad, etc.) and gave us a tour of the ranch and visitors

center. They are looking so good and very happy. The ranch covers 290,000 acres of

land and is owned by the church. It's the largest privately owned ranch in the

world. Six deer came close to us to eat the corn thrown to them and we saw a large

turtle walking by as well as cranes. We had a wonderful time.

Andrea's younger sister, Dana Tollefson, works at Disney World and she sang to

us.....she has an amazing voice!!! She made it possible to get some free tickets as

well as discounts. We also really enjoyed the Marriott vacation Resort that we stayed

at. The kids loved the large pirate ship and I enjoyed the jacuzzi. We found an

alligator in the water close to the land with moss all over it and birds standing on

its head and I think it was fake but Larry said the next day it was gone and he claims

it was real. I think I needed this vacation after a year and a half of battling this

cancer. I feel as if the fight with cancer is over but the money for the medications

and the appointments still lingers on. I had the Torisel infusion yesterday at the

cancer center which takes about 3-4 hours and I still do that weekly for 3 months.

Also, in October I do the Rituxen again. (I do that infusion every 6 months for 4

weeks in a row for the next 2 and 1/2 years) Dr. Kellogg is very pleased with the

results and agreed that there is no sign of cancer or of it coming back. My next

appointment in Texas is in early January at the Burzynski clinic. I want to help

others to be healed as I have been. I am truly blessed by so many friends and family

members. I know that the prayers and faith of others have been heard of God and that

He has blessed me. Life is wonderful!

Seated Dumbbell Presses for Massive Shoulders

This video shows the proper form and benefits of seated dumbbell presses for shoulders. Perform this exercise correctly and you will notice a tremendous improvement in shoulder size, shape and strength.

The keys to this exercise are the following:

1) Use a moderate weight that you can handle for 10 - 12 repetitions.
2) Ascend and descend slowly throughout the movement with no pauses for constant tension.
3) Do not lock out at the top of the movement.

October is Niemann-Pick Disease Awareness Month!

October is the time to raise awareness about Niemann-Pick Disease!

We need to get the word out about this rare disease to doctors, families, government leaders and the general public.

We invite and encourage each family to look within their family structure, friends and community support networks to create their own team and sponsor an awareness campaign and/or fundraising event during October 2010.

The NNPDF has a wealth of experience and resources to support you in any fundraising/awareness project you'd like to undertake, from a simple "Persevere" wristband sale or a "Family and Friends" letter, up to an all-out gala or golf tournament. Take a look at what other NNPDF families are cooking up for October.

One easy and effective way to support the foundation's goals of research into treatments and a cure, and to support families affected by Niemann-Pick Disease, is to participate in this October's Online Auction as a donor and/or a buyer.

It’s a win-win-win situation – the winning bidder gets a great deal on something they like with the added satisfaction of knowing their money is supporting a favorite cause, while the donor has a great feeling knowing their gift will go far to help families affected by NPD. Best of all, the NNPDF is able to use the proceeds to diligently advance our mutual Quest for a Cure!

There are three ways you can help us make this auction a big success: donate an item(s) for auction; bid on auction items; and spread the word to all your family and friends so they can bid, too! (Please visit our Web site for all the details.)

We are busy getting our auction site set up so you can start to "window shop" the items up for sale. Please check the foundation's Online Auction Web page next week for a link to the auction itself. And we hope to hear from you soon regarding what you might be able to donate for this exciting new fundraising opportunity!

As always, Thank You for your continued support! Together, we can make a difference!

2010 Pentchev Research Fellowships Announced

The NNPDF is pleased to announce the recipients of the 2010 Peter G. Pentchev Postdoctoral Research Fellowships!

The research projects of Dr. Nicholas Cianciola of Case Western Reserve University, and Dr. Dorothea Maetzel of the Whitehead Institute for Biomedical Research, were selected through an application process which included reviews by the members of the NNPDF's Scientific Advisory Board (SAB).

Dr. Dan Ory, Chair of the NNPDF's Scientific Advisory Board summarized the two selected projects:

1. Nicholas Cianciola - "Activation of an alternative cholesterol homeostatic mechanism in NPC"
Dr. Cianciola’s project builds upon an exciting observation he made while a graduate student and will explore the role of a viral protein in facilitating movement of cholesterol from lysosomes. The research has the potential to identify new therapeutic targets to stimulate release of cholesterol from lysosomes in the absence of a functional NPC1 protein.

2. Dorothea Maetzel - "In Vitro modelling of Niemann-Pick type C Disease Using Patient-Specific Induced Pluripotent Stem Cells"
Dr. Maetzel’s project will lead to development of pluripotent stem cells from human NPC mutant fibroblasts. The stem cells will then be used for high throughput assays to identify small molecules that can correct the cholesterol accumulation in the NPC cells. Development of these cell lines will allow allow testing of whether the genetic defect can be corrected in the stem cells, which could have therapeutic potential.

For more details, including links to the two researchers' Lay Summaries, please visit the National Niemann-Pick Disease Foundation's Latest Research page.

Congratulations, Dr. Cianciola and Dr. Maetzel! Thank you for your work in advancing Our Quest for a Cure!

Celebration!

I'M IN REMISSION!!!! So exciting. We went to the Burynski Clinic this last week and it official. The blood work from the Gene Therapy Testing at Sonora lab and the ct/pet scan shows NO SIGN of cancer or of it returning.

This week we're heading off to Disney World to celebrate. It's free. We did a trade at work for the tickets and our son Aaron and his wife Andrea are taking care of the hotel and car and the ticket to Disney World and the Epcot Center. We are going to have sooo much fun!

Last night our family came over and our grandson Tate gave me a cinnamon pencil and some shaped and stretchy arm bands as a gift for getting better. He is so cute and has been there for me all along the way. So many people have helped out with money and have exerted their faith through prayer. Thank you to everyone who has helped me!

I return to the Burzynski Clinic in January after another ct/pet scan and they will let me know from there if they will reduce some more of the medicine. They are always very cautious and keep their patients on medicines for awhile after they are in remission. So, my expenses are still $2,600 a month. My neice, Michelle Moyer is helping me out by donating the money from her 3 songs that she put onto itunes for me. So, please check it out. She has an amazing voice.

Also, in November, our family is holding another dinner/auction to raise money for the cause and also to help people to get good deals on Christmas gifts. If you know of any items that can be donated to auction off let us know. It will be a live auction at my sister, Mary Nielson's home.

Life is wonderful and I believe in miracles!!!

New Mexico

The Thomas Family (The family on my side) are having a reunion at the ranch in Blue

water, New Mexico. It's Russell and Mary Nielson's ranch. She's my wonderful

sister. I wasn't sure if we were going to get to go but a check for the gas showed up

at my door and now we are leaving in the morning. It's so nice to have family members

to love and support you when in need. I'm really excited to go and have fun and make

memories with them.

Also, today I received my medicine from the Burzynski Clinic, yeah!!! Just in time. I

really appreciate another family member for paying for them. I know God will bless

them as it was vital for me to continue with this medicine and my care at the clinic.

I'm receiving lots of blessings today. We got an email from our son Thomas in

Washington saying how well he is doing and what a surprise it was to run into his

older brother Aaron at Arby's in Washington. Aaron had a lay over there and was as

surprised as Thomas was to run into him. They got to spend the next day as companions

and had a great visit.

I have 3 grand kids on the way and am so excited and happy to be alive and well and

get to enjoy them. It will make 13 for us. James and Julie came over and set a glass

jar of spagetti sauce on our table. I asked who put it there and went over and picked

it up. It was "Prego" sauce and they had written the words "Julie is" on it. So it

read---Julie is Prego. I said "What? with a weird expression on my face. I was so

excited. When Larry saw it he said "No!" He was also as delighted as me. We were

just so surprised. It was so cute.

I found a wonderful scripture about challenges that I want to share. 2 Corinthians 4:8

"We are troubled on every side, yet not distressed; we are perplexed, but not in

despair." That is exactly how I feel! God is sustaining me and I feel His loving

arms around me every day. I hope that everyone can feel the peace that I have and be

blessed with the things that they need in their lives.

New Product Announcement: Impact Sports ePulse2 Heart Rate Monitor Watch and Calorimeter

Impact Sports Technologies introduces the world's first armband heart rate monitor and calorimeter, the ePulse2™. Until now dynamic heart rate monitors required an uncomfortable chest strap and special watch practically restricting their use to serious athletes. ePulse2, the "People's Fitness Monitor," now makes it practical and convenient for anyone who exercises or is interested in tracking calories burned to obtain the benefits of heart rate monitoring while conducting literally any type of activity from running to walking or simply doing daily chores.

 

Features

 

	Continuous Heart Rate		Max / Min / Avg Heart Rate
	Exercise Calories Burned		Calorie Count Down
	Target Heart Rate Zones		Color Day / Night OLED Display
	Custom Heart Rate Zones		Rechargeable Battery
	Exercise Time		Strapless - Armband Only!
	Stop Watch with Split Times

 

Uses

Since the ePulse2 Armband Heart Rate Monitor does not require an ungainly chest strap and special watch, it can be conveniently used for general fitness, during aerobics, running, tennis/racquetball, golf, resistance training, weight loss monitoring, or even daily activities. Choose a section from the right hand menu to learn more about how monitoring using the ePulse2 can help you!

 

The Technology

The ePulse2 brings the benefits of heart rate monitoring to everyone with its ease of use and convenience. This is accomplishment via sophisticated integration of light-sensor and micro-chip technology (similar to that used in hospital heart rate monitors) that allows the ePulse2 to accurately read the pulse from the forearm under exercise and motion conditions. The ePulse2 is lightweight, incorporates a customizable, easily read day/night full color OLED display and is powered by a rechargeable battery. It requires no programming to immediately display heart rate and with a simple input of personal data, it will also calculate calories burned and target exercise zones.

ePulse2 works equally well for both men and women. This innovative product addresses the prevailing issue other heart rate monitors struggle to overcome...the wearing of a chest strap! At best, chest straps are an annoyance, and at worst, are so uncomfortable that they alienate many people from utilizing this type of valuable training and fitness tool. In particular, women seem very sensitive to chest straps and many won't use heart rate monitors due to this physical requirement. Now, ePulse2 comfortably conveys heart rate (and other important data) allowing anyone to easily regulate their workout intensity and better attain specific fitness or weight management goals.

To order the ePulse2 please visit Unique Fitness Concepts