MORONI A Utah toddler is one of the first in the U.S. to receive a potentially groundbreaking therapy, and his father is doing everything he can to give his son the strength to win his fight.
Deep in the farm country of central Utah, a family is counting their blessings.
Yes, I would call it living the dream, yes maam, said Alex Wight, who lives with his family in Moroni.
Four years ago, while riding in the high country, Wight and his horse went off a cliff. The fall shattered Wights foot. Far from help, I was definitely going into some shock and I thought, Man, Im never going to get out of here,' Wight said.
Wight climbed back on that horse and rode for 20 miles. If I would have quit, I would have never made it out, he said.
Never was that toughness more vital than when his wife gave birth to their son, Cinch. I was pretty torn up. I cried a lot. Not gonna lie, Wight said.
Newborn screenings showed Cinch has spinal muscular atrophy, or SMA.
I cried. I was working at the sawmill, didnt even tell anyone I was leaving, (I) just put my machine away and left, Wight said.
The neuromuscular disorder paralyzes a baby in the first few weeks or months of life.
My first thought was that my son will never be able to ride broncs, rope, or just be a cowboy in general, Wight said.
Many babies with SMA dont survive to their second birthday, but the day after Cinch was born, the U.S. Food & Drug Administration approved a new therapy. We felt like our prayers were answered, Wight said.
In a single infusion, a virus delivers a new copy of the gene into the babys nerves, said Dr. Russell Butterfield, a pediatric neurologist with University of Utah Health and Intermountain Primary Childrens Hospital.
Only this time, its not delivering a diseased gene that just causes more virus to be made, but a gene that we care about, Butterfield said.
Its a $2.1 million treatment.
I was thinking, Man, theres no way Ill ever pay for it, Wight said.
Wight was determined. We just stayed on top of it. I wasnt going be the one to not get it, he said.
With help from insurance and Primary Childrens Hospital, Cinch got the gene replacement therapy through the Primary Childrens Center for Personalized Medicine.
The Wights wouldnt know for months if it worked. Amber Wight, Cinchs mother, said, Every days a waiting game, we have no idea.
Alex wrote a book to teach children they can do hard things, even when theyre hurt or discouraged.
Early one morning, a cowboy stepped out of his camp and into the corral, Wight said, reading the book to his son.
He held his breath the day Cinch took his first steps. He just cruises, Wight said.
It just brings a tear to your eye, to be honest, Butterfield said.
Time will tell if the gene replacement therapy is indeed a breakthrough. I just hope he never quits, Wight said.
With love wider than a prairie I guess on a scale of one to 10, Id call it a 12, Wight said and with the wonders of medical science, Cinch has a shot at a bright future, full of promise.
You can get Alex Wights book at Amazon.com.
Read the original post:
Utah toddler one of first to receive groundbreaking gene replacement therapy - KSL.com
- Gene Therapy for Genetic Disease: The Long and Winding Road [Last Updated On: August 17th, 2024] [Originally Added On: October 10th, 2011]
- Gene Doping: Super Athletes in 2008 Beijing Olympics - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 14th, 2011]
- The 3 Rs of DNA: Molecules to Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 15th, 2011]
- BIT 3.1 Entrez Gene - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 15th, 2011]
- Professor Ian Everall - Gene expression in schizophrenia - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 16th, 2011]
- Gene Therapy Promising Treatment (Part 2 of 4) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 16th, 2011]
- Cancer Research @ NC State's Centennial Biomedical Campus - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 16th, 2011]
- Gene Therapy Promising Treatment (Part 1 of 1) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 19th, 2011]
- HiTech_U3L3_Biotech in Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 19th, 2011]
- Sri Sathya Sai Medical Camps Worldwide - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 20th, 2011]
- Dr. Joseph M. DeSimone: 2008 Winner, $500000 Lemelson-MIT Prize - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 21st, 2011]
- Non-Hodgkin's Lymphoma Cancer Survivor Testimony - (Sanoviv Medical Institute) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 22nd, 2011]
- Vaccines: Medicine for the Healthy - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 24th, 2011]
- For Your Health - Health Care Informatics - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 24th, 2011]
- A Century of Stem Cells - Johns Hopkins Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 24th, 2011]
- Functional Repair of Human Donor Lung by IL-10 Gene Therapy (EX VIVO Lung) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 25th, 2011]
- Science Talk: Vaccine Made With Synthetic Gene Protects Against Deadly Pneumonia - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 25th, 2011]
- Health Matters: Family Medicine - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 26th, 2011]
- Dr. Jeffrey Myers Explains Genetic Variations in Head and Neck Cancer - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- The Emerging Network of Data for Understanding the Interactions of Genes and Drugs - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Burzynski_ Cancer Is Serious Business Part 4/8. wmv - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Pt2: Do you understand the new genetics? Maybe you don't have that gene! - Andrew Shelling - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Alien Genetic Takeover of Humanity! - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Burzynski | 2-DVD Set Extended Edition Montage Preview of New Material | Cancer Is Serious Business - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Genes, Genomes and the Future of Medicine - Richard Lifton, MD, Ph.D. - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Burzynski The Movie ~ Cancer Is Serious Business ~ High Quality - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Designing Life for Mars Colonization (Brainstorm Ep20) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- Dedication Event: Santa Monica UCLA Medical Center [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- DR. Burzynski The Movie - Cancer Is Serious Business (PT.1) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 27th, 2011]
- burzy - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Alien Genetic Takeover: The End of Humanity - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Burzynski_ Cancer Is Serious Business Part 1/8 .wmv - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Mike Schmidt - ECMO Patient - Penn State Hershey Medical Center - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Disrupting the HIV Virus (Brainstorm Ep24) - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Diagnosis and Treatment of Hepatorenal Syndrome - Moving Targets? - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Big Bucks, Big Pharma: Marketing Disease and Pushing Drugs - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Roles of RNA in Gene Regulation - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Diatom PCR Colony Screen - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Kleanthis G. Xanthopoulos, Ph.D., CEO, Regulus, talking on the impact of microRNA medicines - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Burzynski_ Cancer Is Serious Business Part 2/8 .wmv - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 28th, 2011]
- Sickle Cell Anemia: Stem Cell Gene Therapy - A Patient's Perspective - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Gene-based lung cancer treatment video - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- HIV Resistant Genes...Rhesus Negative, Excess PK [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Futures in Biotech 83: Bioinformatics: Essential Gene names Skewed in a Network of Blame - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 29th, 2011]
- Keeping Blood Young, Fighting Sickle Cells (Brainstorm Ep28 - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- News@Northwestern - NUGENE - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- UK Medical Student Mary Burchett's Research is Personal - Video [Last Updated On: August 17th, 2024] [Originally Added On: October 31st, 2011]
- Cannabis 4 Crohn's: 2 Years As Medical Marijuana Patient, Dr Mind Bender [Last Updated On: August 17th, 2024] [Originally Added On: November 8th, 2011]
- Medical Haptical Robot for the Eye - Eye-Rhas, - TU Eindhoven - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 8th, 2011]
- The Ghost in Your Genes - Horizon - BBC - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 9th, 2011]
- The Ghost in Your Genes - BBC Horizon - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- Burzynski: Cancer Is Serious Business (Trailer) - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 16th, 2011]
- Genes, Ancestry, and Personalized Medicine with Dr. Michael Kane - Interview Part 1.mp4 - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 25th, 2011]
- The Suicide Gene - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 29th, 2011]
- Ard Louis on Dawkins and Selfish Genes - Video [Last Updated On: August 17th, 2024] [Originally Added On: November 30th, 2011]
- TEDxLex - Dr. Alicia L. Bertone - Gene Jockeying for a Win in Regenerative Medicine.mp4 - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 3rd, 2011]
- Introduction to Gene Network - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 7th, 2011]
- The demise of a dogma. The deaf somatic gene listens - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 7th, 2011]
- Future of health care, hospitals, medicine and wellbeing - by Dr Patrick Dixon - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 8th, 2011]
- Electro-Medicine : Biological Physics : Neurological Research Advancements - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 12th, 2011]
- Haemophilia gene therapy breakthrough - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 15th, 2011]
- Duke researchers: Gene discovery explains how fruit flies retreat from heat - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 16th, 2011]
- Focus on Nikolaus Rajewsky - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 18th, 2011]
- Deadly Monopolies Medical Ethicist Harriet Washington on How Firms are Taking Over Life Itself - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 23rd, 2011]
- Open fetal surgery: past, present and future - Video [Last Updated On: August 17th, 2024] [Originally Added On: December 23rd, 2011]
- The Human Genome and Individualized Medicine - David Valle, MD - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 14th, 2012]
- Environmental Contributors to Autism Spectrum Disorders - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 20th, 2012]
- When Cancer Runs in the Family - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 20th, 2012]
- Genome Enabled Electronic Medical Record (GenE EMR) - William Knaus - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 22nd, 2012]
- Nuvigil withdrawal - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 23rd, 2012]
- Cancer Cure Documentary - Dr. Burzynski Antineoplaston Therapy - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 26th, 2012]
- Next Generation Cancer Gene Sequencing for Clinically Actionable Mutations - Video [Last Updated On: August 17th, 2024] [Originally Added On: January 26th, 2012]
- Fibrocell Science, Inc. Announces Recognition for LAVIV™ (azficel-T) at The 2012 Cell & Gene Therapy Forum, Washington ... [Last Updated On: August 17th, 2024] [Originally Added On: January 31st, 2012]
- Gene Mutation Linked to Inappropriate Lipid Buildup in Liver [Last Updated On: August 17th, 2024] [Originally Added On: February 1st, 2012]
- 'Goldilocks' gene could determine best treatment for tuberculosis patients [Last Updated On: August 17th, 2024] [Originally Added On: February 3rd, 2012]
- James Wilson, MD, Ph.D., on Gene Therapy as a Disruptive Technology - Video [Last Updated On: August 17th, 2024] [Originally Added On: February 3rd, 2012]
- 'Goldilocks' gene used to find drug treatment that is 'just right' for TB patients [Last Updated On: August 17th, 2024] [Originally Added On: February 4th, 2012]
- Gene therapy, a totally Italian success - Video [Last Updated On: August 17th, 2024] [Originally Added On: February 4th, 2012]
- Electro-Medicine : Neurons function revealed - Video [Last Updated On: August 17th, 2024] [Originally Added On: February 4th, 2012]
- Gene mutation discovery sparks hope for effective endometriosis screening [Last Updated On: August 17th, 2024] [Originally Added On: February 7th, 2012]