Texas Family Fights to Access $2.1 Million Treatment for Baby – NBC 5 Dallas-Fort Worth
By daniellenierenberg
A Texas family said they faced a fight for their childs life after their daughter's health plan provider denied coverage of a medication known as the most expensive drug in the world.
The family said doctors told them the one-time infusion that costs millions of dollars was their daughters best hope.
Read on to learn more about their story and how an "army" of strangers stepped in.
At 4-months old, every move Aniya Porter makes is a miracle to her parents.
Aniya Porter was born with a rare genetic disease called spinal muscular atrophy or SMA.
You go day-by-day. Is my daughter going to stop breathing? Is she going to stop attempting to stand up? Is she going to stop putting her head up? Will Porter said.
Aniya was born with a rare genetic disease called spinal muscular atrophy or SMA.
SMA progressively kills motor neurons, the nerve cells in the brain stem and spinal cord that control essential functions like talking, walking, swallowing and breathing.
We live every day wondering and hoping that nothing else happens to her, that she doesn't lose any more of her motor neurons, said Hailey Weihs, Aniyas mother.
There is treatment.
In 2019, the FDA approved a drug called Zolgensma which carries a price tag of $2.1 million. The one-time gene therapy, given through an IV, is designed to replace the function of a missing or non-working SMN1 gene in a patient to stop the progression of SMA preserving motor neurons before theyre gone.
It doesn't give back what she has already lost. That's why it's so time-sensitive, explained Weihs.
When Aniyas doctor prescribed Zolgensma, Weihs and Porter said their daughters insurance, a Medicaid provider in Texas, denied coverage of the drug.
She got the denial from her insurance and that was devastating for us, said Weihs.
As they appealed, they said Aniya started to show signs she may lose control of her muscles.
She started to stop having reflexes in her legs and then her tongue started twitching, said Porter.
Racing against time, Weihs connected with other families of kids with SMA and a lawyer who agreed to represent Aniya for free.
Aniyas attorney, Eamon Kelly, told NBC 5 Responds, All the doctors agree that Aniya Porter should receive this treatment.
Kelly, who is based in Chicago, said Aniya is the seventh child with SMA hes represented in insurance coverage battles.
In Aniyas case, he said the health plan provider said the treatment was not medically necessary because doctors believe Aniya has four copies of the SMN2 gene, also known as a backup gene, which can indicate a milder illness.
Kelly argued backup genes, along with other available therapies, would not be enough to keep Aniya from losing muscle function. However, he said cutting-edge gene therapy could help.
We have a treatment that will take a little girl that is going to have a degenerative disease that threatens her life, that will put her in a wheelchair and as long as we get it to her before she's two years old and before she loses her motor neurons, she'll walk, she'll dance, she'll live a full life. Its like science fiction, Kelly said.
The first child with SMA Kelly represented is Maisie Forrest, who received Zolgensma in 2019 when she was 20 months old.
Ciji Green, Maisies mom, said Maisie was on a ventilator 22 hours a day before getting the drug.
We met with her pulmonologist and I just wept, Green recalled. I told him she's not going to make it to two and he didn't offer any words of hope because he knew Maisie was on the decline as well.
Maisie is now 4 years old.
She touches my face and it's absolutely beautiful, said Green.
Maisie is playing, talking and crawling.
Something that I have now that I didn't have was hope, hope that I will get to see her continue to meet milestones that she was never supposed to meet, Green added.
Maisies mom and a team of volunteers known as Maisies Army introduced Aniyas parents to Eamon Kelly. Last month, Kelly represented Aniya at a Medicaid State Fair Hearing.
Aniyas family requested the hearing from Texas Health and Human Services.
A week after the hearing and a few days after NBC 5 reached out to Aniyas health plan provider, Superior HealthPlan, Superior told Aniyas family Zolgensma would be covered for Aniya.
We have fought for four months. Those have been the hardest four months of our lives, said Weihs.
On April 27, Aniyas family made the trip from their home in Abilene to Cook Childrens Medical Center in Fort Worth where Aniya received the drug.
She's going to sit up on her own, she is going to walk one day, she's going to feed herself with a spoon, she's going to walk down and she's going to get her diploma, Weihs said.
NBC 5 Responds reached out to Superior HealthPlan by phone and email. We didnt hear back.
We asked Texas Health and Human Services about options for families denied coverage for Zolgensma.
It told us, in part, Medicaid covers medically necessary services including medications, and those services are delivered through managed care organizations. If the prior authorization is denied, the provider or the member can appeal the decision and MCOs have flexibility to make medically necessary decisions. Members also have the right to access the State Fair Hearing process with or without an External Medical Review (EMR). The EMR is conducted by a third-party Independent Review Organization.
It also shared, Medicaid covers alternative therapies for spinal muscular atrophy treatment, including Spinraza (nusinersen) and Evrysdi (risdiplam). Also, some manufacturers offer patient assistance programs.
A spokesperson for Novartis, which makes Zolgensma, told NBC 5, in part, Zolgensma (onasemnogene abeparvovec) is a transformative and highly innovative gene therapy for a devastating, progressive genetic disease. This one-time gene therapy is priced based on the value it provides to patients, caregivers and health systems.
It also said, Novartis is working in partnership with governments and health care systems worldwide to identify and define new sustainable access models.
Aniyas parents said she will still see a doctor and be monitored after getting the gene therapy treatment. Theyre hopeful about her prognosis.
We just knew we couldnt give up, Weihs said.
Weihs tells NBC 5 Responds the family is now focused on helping other kids get access to a drug they believe is priceless.
I don't care how expensive it is. It's a child's life. Every baby deserves a chance, said Porter.
Novartis said more than 1,800 patients have been treated with Zolgensma worldwide.
Texas newborn screening program began screening for SMA last June. The states health and human services website reports SMA is among the leading genetic causes of death among infants and toddlers.
NBC 5 Responds is committed to researching your concerns and recovering your money. Our goal is to get you answers and, if possible, solutions and a resolution. Call us at 844-5RESPND (844-573-7763) orfill out our customer complaint form.
Read the original here:
Texas Family Fights to Access $2.1 Million Treatment for Baby - NBC 5 Dallas-Fort Worth
- Bone marrow mesenchymal stem cells modulate miR-202-3p to suppress neuronal apoptosis following spinal cord injury through autophagy activation via... - December 9th, 2024
- Stem Cells Reveal Secret to Beneficial Proteins for mRNA Therapy - An Interview with Neuroscientist Prof. Dr. Antal Ngrdi - Szegedi Tudomnyegyetem - December 9th, 2024
- Much-anticipated human trial aiming to repair spinal cord damage about to begin - ABC News - October 21st, 2024
- The Science Of Health: Are Spinal Cord Injuries Irreversible? Know Science Advances That Can Cure Them In The Future - ABP Live - October 16th, 2023
- Evaluating the Growth Prospects of the Global Nerve Repair & Regeneration Market at a CAGR of 6.5% | Emergen - EIN News - April 21st, 2023
- Regenerative Therapies Market is Set to Grow at a CAGR of 8.7% by 2033, Propelled by Advancements in - EIN News - March 17th, 2023
- Kadimastem Submits IND Application to the FDA for its Phase IIa Clinical Trial with AstroRx for the Treatment of ALS - Marketscreener.com - February 21st, 2023
- My Back Is All F*cked Up 55-Year-Old Joe Rogan Curses at Worst Jiu-Jitsu for Painful Health Condition - EssentiallySports - February 21st, 2023
- Brain and Spinal Cord Tumors: Hope Through Research - January 3rd, 2023
- 14.3 The Brain and Spinal Cord Anatomy & Physiology - January 3rd, 2023
- Stem Cell Therapy for Spinal Cord Injury - PubMed - January 3rd, 2023
- Spinal cord injury - Diagnosis and treatment - Mayo Clinic - December 25th, 2022
- Spinal Cord Injury: Hope Through Research | National Institute of ... - December 1st, 2022
- Stem cell controversy - Wikipedia - October 13th, 2022
- Stem Cells Australia | Australian research, stem cell treatments and ... - October 13th, 2022
- The eye and stem cells: the path to treating blindness - October 13th, 2022
- World's first stem cell treatment for spina bifida delivered during fetal surgery - UC Davis Health - October 13th, 2022
- Fighting One Disease or Condition per Day - Daily Kos - October 13th, 2022
- UPDATE: NurExone Signs Letter of Intent with Nanometrix for Its Exosome and Cargo Molecular Profiling AI-Driven Technology - Yahoo Finance - October 13th, 2022
- Global Cell Therapy Market Report (2022 to 2028) - Featuring Thermo Fisher Scientific, MaxCyte, Danaher and Avantor Among Others -... - October 13th, 2022
- Horizon Therapeutics plc Announces New UPLIZNA (inebilizumab-cdon) Data in Neuromyelitis Optica Spectrum Disorder (NMOSD) to be presented at ECTRIMS... - October 13th, 2022
- Physiology, Spinal Cord - StatPearls - NCBI Bookshelf - October 5th, 2022
- IMAC Holdings, Inc. Announces Completion of Third Cohort of its Phase 1 ... - October 5th, 2022
- Revolutionary Jab that Could Repair Spinal Cord Injuries Developed by Scientists - Good News Network - October 5th, 2022
- How the 'Love Hormone' Oxytocin May Help Heal Heart Muscles - Healthline - October 5th, 2022
- Unlocking the Mysteries of Brain Regeneration Groundbreaking Study Offers New Insight - SciTechDaily - October 5th, 2022
- In Conversation: How to understand chronic pain - Medical News Today - October 5th, 2022
- New drug could cure aggressive brain cancer stopping tumours in their tracks... - The US Sun - September 27th, 2022
- Rehabilitating spinal cord injury and stroke with graphene and gaming - Nanowerk - September 19th, 2022
- Induced Pluripotent Stem Cells Market Reaches at a CAGR of 8.0% in the Forecast Periods [2021-2031] - BioSpace - September 19th, 2022
- Axolotls can regenerate their brains - Big Think - September 19th, 2022
- IMAC Holdings, Inc. Announces Completion of Third Cohort of its Phase 1 Clinical Study of Umbilical Cord-Derived Mesenchymal Stem Cells for the... - September 11th, 2022
- Spinal Muscular Atrophy: Causes and importance of early diagnosis for proactive management - Firstpost - September 11th, 2022
- Increasing Road Accidents and Fall Injuries among Aged Population Primarily Driving Need for Orthopedic Navigation Systems: Fact.MR Analysis - Yahoo... - September 3rd, 2022
- Culture of human nasal olfactory stem cells and their extracellular vesicles as advanced therapy medicinal products - Newswise - August 10th, 2022
- Curious kids: what is inside teeth? - The Conversation - August 10th, 2022
- Human placental mesenchymal stem cells derived exosomes improved functional recovery via attenuating apoptosis and increasing axonal regeneration... - August 2nd, 2022
- How the Regenerative Properties of Glioblastoma Can Be Terminated - Gilmore Health News - August 2nd, 2022
- New TSXV listing looks to address the $3B spinal cord injury treatment market (NRX.V) - FXStreet - July 25th, 2022
- Human iPSC co-culture model to investigate the interaction between microglia and motor neurons | Scientific Reports - Nature.com - July 25th, 2022
- Negligence in treatment of diseases like glioblastoma can be fatal, seminar told - The News International - July 25th, 2022
- What lab-grown cerebral organoids are revealing about the brain - New Scientist - July 25th, 2022
- Innovative Therapies, Care Equity Highlight 2022 ASCO Annual Meeting - Targeted Oncology - July 16th, 2022
- Global Stem Cell Manufacturing Market Value Projected To Reach USD 21.71 Billion By 2029, Registering A CAGR Of 9.1% - Digital Journal - July 16th, 2022
- Stem Cell Therapy Market Is Expected To Reach USD 455.61 Billion By 2027 At A CAGR Of 16 percent By Forecast 2027 Says Maximize Market Research (MMR)... - June 30th, 2022
- This startup wants you to have a personal stem cell stash - Freethink - June 30th, 2022
- Parents of 12-Year-Old Boy Praying for a Miracle, Appealing UK Judge's Decision to Remove Life Support - CBN.com - June 30th, 2022
- The end of Roe v. Wade affects more than just abortion - Vox.com - June 30th, 2022
- Horizon Therapeutics plc Submits Regulatory Filing for UPLIZNA (inebilizumab) in Brazil - Business Wire - June 20th, 2022
- Effect of Electrical Stimulation on Spinal Cord Injury: In Vitro and In Vivo Analysis - Newswise - June 11th, 2022
- First-of-its-Kind Stem Cell and Gene Therapy Highlighted at Annual Stem Cell Meeting - Newswise - June 11th, 2022
- UK Judge to Decide if 12-Year-Old Will Be Removed from Life Support, Parents Beg for More Time to Heal - CBN.com - June 11th, 2022
- 'This is my life, and I'll try anything to save it': Woman with MS raising funds for treatment - The Brandon Sun - May 29th, 2022
- Racing Thoughts: Quadriplegic Man Drives Race Car With His Brain - Newsy - May 29th, 2022
- Physical therapy for vertigo: Exercises, benefits, and more - Medical News Today - May 29th, 2022
- Researchers find new function performed by almost half of brain cells - Medical News Today - May 13th, 2022
- Severe COVID-19 may cause cognitive deficits equivalent to 20 years of aging - Medical News Today - May 13th, 2022
- Stem Cell Magic: 5 Promising Treatments For Major Medical Conditions - Study Finds - April 29th, 2022
- Neural Stem Cell Therapy For Spinal Cord Injury To Tap Into The Potential Of Stem Cells - Optic Flux - April 15th, 2022
- Still Blooming: Sams mission to raise money for spinal cord injury research - 7NEWS - April 15th, 2022
- Lineage and Cancer Research UK Announce Completion of Patient Enrollment in Phase 1 Clinical Study of VAC2 for the Treatment of Non-small Cell Lung... - April 15th, 2022
- Lineage Announces Pipeline Expansion to Include Auditory Neuronal Cell Therapy for Treatment of Hearing Loss - Galveston County Daily News - March 22nd, 2022
- COVID-19: Even mild to moderate infection may cause brain anomalies - Medical News Today - March 22nd, 2022
- Scots mum with MS says 50k treatment abroad is 'last hope' of halting disease - Daily Record - January 18th, 2022
- Mending the gap: U of T's Molly Shoichet joins team developing new treatments for spinal cord injuries - News@UofT - January 18th, 2022
- Spinal Cord Injury Information Page | National Institute ... - January 3rd, 2022
- Dancing molecules successfully repair severe spinal cord ... - January 3rd, 2022
- Best 2021 Medical Breakthroughs And Treatments to Beat Cancer, Alzheimer's, Diabetes & More - Good News Network - January 3rd, 2022
- Global Regenerative Medicine Market is Expected to Reach USD 57.08 Billion by 2027, Growing at a CAGR of 11.27% Over the Forecast Period. -... - December 23rd, 2021
- Scientists unravel a gene function that helps the genesis of neurons - Research Matters - December 23rd, 2021
- The 10 Most Compelling Research Stories of 2021 PharmaLive - PharmaLive - December 23rd, 2021
- 2021: The year in review | YaleNews - Yale News - December 23rd, 2021
- Polymyositis Pipeline to Progress with New and Emerging Drugs for Treatment, Analyzes DelveInsight - GlobeNewswire - December 10th, 2021
- Cell and Gene Therapy Market to reach US$ 47,095.2 Mn by end of 2028, Says Coherent Market Insights - PRNewswire - November 22nd, 2021
- From asthma to cancer to infertility, the new treatments, jabs and meds making us healthier... - The Sun - November 22nd, 2021
- Improving motor neuron-like cell differentiation of hEnSCs by the combination of epothilone B loaded PCL microspheres in optimized 3D collagen... - November 8th, 2021
- Akiko Nishiyama Explains the Many Strengths of a Degree in Physiology and Neurobiology - UConn Today - UConn Today - October 28th, 2021
- Team finds way to enhance stem cell therapy for CNS injuries - BioPharma-Reporter.com - October 28th, 2021
- 'Rogue' antibodies found in brains of teens with delusions and paranoia after COVID-19 - Livescience.com - October 28th, 2021
- Traumatic Spinal Cord Injury: An Overview of ... - October 16th, 2021