Mumbai: Baby with genetic disorder needs Rs 16 crore therapy to live – Mid-day
Posted: December 12, 2020 at 7:56 pm
The COVID-19 lockdown had brought some cheer for Andheri-based Kamat couple as they welcomed their first child - Teera - on August 14. But soon things went awry when their daughter was diagnosed with a rare genetic disorder - Spinal Muscular Atrophy (SMA). Three-month-old Teera's parents - Mihir, 35, and Priyanka Kamat, 33, have been doing everything possible for their daughter's treatment but the biggest challenge for them is to raise '16 crore for a single shot of gene therapy, the only way she can survive.
Teera with her parents Mihir and Priyanka Kamat
Teera has been diagnosed with SMA Type 1, which is said to be a more serious form of the disorder. Speaking to mid-day, Mihir, who works for a software company, said, "Teera was born a happy and cheerful baby. As weeks went by, we noticed she was having heavy breathing patterns and it would also take a long time to feed her. Very soon, she developed multiple issues like severe dehydration, heavy breathing, choking on feeds etc, and we finally found a doctor in our area who recommended we start bottle feeding."
"On the day of her vaccination (September 30), her doctor noticed that she was choking on her first drops of vaccine. She was also not being able to resist with her hands and the right leg wasn't moving," Mihir recalled.
"The doctor referred us to a paediatric neurologist who mentioned that Teera might have hypotonia, slow reflexes, paradoxical respiration and a weak cry. Following her EMG and genetic test, we got to know that she is suffering from SMA Type 1. The next shock was the cost of the drug, which needs to be imported from the United States. Teera needs it before she turns six months old, the golden period to save her. We started doing a bit of research on the disorder and found out about some foreign nationals who have written positively about the treatment and how they could 'crowd fund' such a large sum by registering on a similar platform," added Mihir.
"We registered our daughter's details with crowd funding platform Impact Guru and in less than a month 8,187 donors contributed nearly '2.36 crore, said the couple. Mihir further said, "Our consulting paediatric neurologist Dr Neelu Desai has already registered Teera for the Switzerland HQ Novartis pharma company global lottery, where selected registered patients get the drug free of cost." "Just three weeks ago Teera had to be rushed to Holy Spirit hospital in Andheri East, as she got choked during her feed and turned pale. She had to be kept at the hospital for the next couple of days," her father said.
SMA is a progressive, rare genetic disease that is caused by missing genes or those that don't work properly, which might be survival motor neuron 1 (SMN1). This gene is basically the blueprint responsible for making a protein required for the survival of the spinal nerve cells. Without this SMN gene the cells in the body start to die and motor neuron cells become weaker thus causing muscle weakness in every part of the body below the neck.
Piyush Jain, co-founder and CEO of ImpactGuru.com, said, "In one month, 8,187 donors have contributed '2.36 crore. This is Impact Guru's highest single fundraiser. Among the donors is a noted Bollywood actor, who donated '5 lakh We are currently working towards ensuring the target is met by all means."
Dr Neelu Desai
Dr Desai said, "Teera is one of the youngest patients I'm treating at the moment. If she doesn't get the special gene therapy, then she might survive for about two years. Until last five years, there was no treatment for SMA, but now we have gene therapy, for which Teera has been registered."
She further said, "This is a rarest of rare disorder (1 in 5,000 infants suffer from it world over). In case a baby does not develop proper motor skills or his/her body feels very soft then parents should consult paediatricians. Parents who have a child with SMA, have 25 per cent chances of the same disorder developing in the subsequent pregnancies, which can be confirmed in the first few months by doing a genetic test."
Link to the fundraiser - https://www.impactguru.com/fundraiser/donate-to-teera
Keep scrolling to read more news
Catch up on all the latest Crime, National, International and Hatke news here. Also download the new mid-day Android and iOS apps to get latest updates.
Mid-Day is now on Telegram. Click here to join our channel (@middayinfomedialtd) and stay updated with the latest news
See the article here:
Mumbai: Baby with genetic disorder needs Rs 16 crore therapy to live - Mid-day
- After the Nobel, what next for Crispr gene-editing therapies? - The Guardian - February 25th, 2021
- Lucky winner! Rs 16 crore lottery gives new lease of life to 14-month-old Fatima - Zee Business - February 25th, 2021
- 14 Emerging Tech Trends for 2021and Dallas-Area Companies That Are Innovating for the Future Dallas Innovates - dallasinnovates.com - February 25th, 2021
- Gene Therapy - Cleveland Clinic - February 19th, 2021
- 14-month-old lottery winner with killer disease gets new life after expensive therapy in Bengaluru - India Today - February 17th, 2021
- 16 crore drug is the hope for SMA patients - The Hindu - February 14th, 2021
- The First Gene Based Project to be conducted in the Rio Grande Valley - Mega Doctor News - February 14th, 2021
- Spondyloarthritis: Types, symptoms, treatment, and more - Medical News Today - February 14th, 2021
- MSI-High and Colon Cancer: What It Means, Treatment Options, and More - Healthline - February 6th, 2021
- Targeted therapy for lung cancer: Side effects and more - Medical News Today - February 6th, 2021
- Baby can be saved from rare genetic disorder - but sister, 5, will die of it - Metro.co.uk - January 31st, 2021
- ALK Positive Lung Cancer: Life Expectancy, Survival, and More - Healthline - January 31st, 2021
- Dr. William Kelley inducted into IAOTPs Hall of Fame - PRUnderground - January 31st, 2021
- Last Call with Jenna Balestrini, the WPI grad treating cancer with cell therapy - Worcester Mag - January 14th, 2021
- Province gives family facing $2.8M drug bill a glimmer of hope - CBC.ca - January 14th, 2021
- New Combination Therapy Tested By Children's May Offer Hope For Leukemia Patients - WVXU - December 22nd, 2020
- This 6-year-old Iowa boy is losing his vision and hearing. But his family holds on to hope. - The Gazette - December 22nd, 2020
- What Is Gene Therapy? How Does It Work? | FDA - December 17th, 2020
- What is gene therapy? - The Star Online - December 17th, 2020
- What It's Like Rising A Little Boy With Advanced Breast Cancer - Oprah Mag - December 17th, 2020
- EAPM Advancing gene therapy with Advanced Therapy Medicinal Products - EU Reporter - December 16th, 2020
- San Diego's Locanabio raises $100 million for treatments aimed at degenerative diseases - The San Diego Union-Tribune - December 16th, 2020
- Italy needs new restrictions to avoid third, devastating COVID-19 wave - PM to paper - EU Reporter - December 16th, 2020
- Yes, men can get breast cancer here are the symptoms to watch for - Insider - INSIDER - December 12th, 2020
- Gene Therapy Liberates Hemophilia B Patients from Requiring Regular Infusions of Clotting Factor - MedicalResearch.com - December 12th, 2020
- Vaccination in the Era of Covid - Touro College News - December 7th, 2020
- Genentech Announces New Data Reinforcing the Long-Term Benefit of Venclexta-Based Combination for People With Relapsed or Refractory Chronic... - December 7th, 2020
- Medicare and cystic fibrosis: Coverage, options, treatments, and costs - Medical News Today - December 7th, 2020
- The FDA has approved the first drug to treat the rapid-aging disease progeria - Science News - November 26th, 2020
- 'This is why I'm here': A Detroit Lions VP tries to save her daughter from rare disease - ESPN - November 24th, 2020
- Encouraging Clinical Data for Controlled IL-12 for the Treatment of Glioblastoma and DIPG - OncoZine - November 24th, 2020
- SwanBio Therapeutics Expands Board of Directors with Appointments of Proven Industry Leaders - Business Wire - November 24th, 2020
- SwanBio Therapeutics Expands Board of Directors with Appointments of Proven Industry Leaders - BioSpace - November 23rd, 2020
- Rare Mutation in the PCSK9 Gene Confers Long Healthy Life - MedicalResearch.com - November 23rd, 2020
- Living through the second wave - Pakistan Today - November 23rd, 2020
- How two blind brothers became Two Blind Brothers for a cause - The Columbian - November 10th, 2020
- How two blind brothers created a one-of-a-kind shopping experience to raise money for a cure - WXII The Triad - November 10th, 2020
- The ethical way to alter organisms - The Boston Globe - November 10th, 2020
- Teladoc Is A Strong Buy: A Radical Healthcare Change Will Come - Seeking Alpha - November 10th, 2020
- Medical Milestone In Boston: 13-Year-Old Gets New Gene ... - November 5th, 2020
- How immunotherapy is revolutionizing cancer care - Genetic Literacy Project - October 29th, 2020
- The good news about breast cancer - The Gazette - October 29th, 2020
- Harris County parents win insurance appeal for 8-year-olds MD treatment - WRBL - October 29th, 2020
- Biohack the ageing process by taking care of this little protein in your body - Lifestyle Asia - October 22nd, 2020
- 'I have faith and I prayed a lot' - News from southeastern Connecticut - theday.com - October 22nd, 2020
- Piqray Improves Overall Survival for Advanced-Stage Hormone-Receptor-Positive, HER2-Negative Breast Cancer With PIK3CA Mutation - Breastcancer.org - October 20th, 2020
- Beating Cancer: Prevention must be 'cornerstone' of EU's new approach - The Parliament Magazine - October 20th, 2020
- Watch: New 5-minute injection a game-changer in breast cancer therapy in UAE - Gulf News - October 20th, 2020
- Homer Glen Mom and Nurse Breast Cancer-Free after Radiation Therapy Clinical Trial at UChicago Medicine Comprehensive Cancer Center at Silver Cross -... - October 20th, 2020
- Teenager among first to receive gene therapy treatment for rare condition - KRDO - October 8th, 2020
- Proposition 14: With Just Handful of Cures, California Stem Cell Agency's Fate Is In Hands of Voters - KQED - October 8th, 2020
- Teenager among first to receive gene therapy treatment for rare condition - NewsChannel 3-12 - KEYT - October 7th, 2020
- Greenbrier County teen to be among first in nation to receive gene therapy for Hurler's Syndrome - WVVA TV - October 7th, 2020
- Regenxbio RGX-121 Clinical Program, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha - October 7th, 2020
- Women In Longevity Medicine And The Rise Of The Longevity Physician - Forbes - October 7th, 2020
- Timothy Ray Brown, First Patient to Be Cured of HIV, Dies of Leukemia - BioSpace - October 7th, 2020
- Different Types of Childhood Cancer | INTEGRIS - Integris - October 4th, 2020
- Gene therapy company Taysha completes sprint from first funding to IPO - BioPharma Dive - September 26th, 2020
- Focused on the future: Innovative college programs in N.J. pave way for the real world - Jersey's Best - September 24th, 2020
- Burosumab Is a 'Game Changer,' Effective in All Subgroups of XLH - Medscape - September 22nd, 2020
- 10 Forgotten Spiderman Sidekicks They Need To Bring Back | CBR - CBR - Comic Book Resources - September 22nd, 2020
- Genentech Presents New Data From Multiple Phase III Studies of Tecentriq in Triple-Negative Breast Cancer at ESMO Virtual Congress 2020 - Business... - September 21st, 2020
- BCS Announces Partnership With 2nd.MD to Improve Member Healthcare Cost, Quality, and Outcomes - PRNewswire - September 19th, 2020
- Novus Therapeutics Announces Acquisition of Anelixis Therapeutics - Business Wire - September 19th, 2020
- Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal - September 8th, 2020
- CRISPR can help combat the troubling immune response against gene therapy - The Conversation US - September 6th, 2020
- How Is Ovarian Cancer Diagnosed? Everything You Need To Know - NDTV Doctor - September 6th, 2020
- Genentech Announces FDA Approval of Gavreto (pralsetinib) for the Treatment of Adults With Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer |... - September 6th, 2020
- Gilmore Health: A Q&A Session on Genetic Diseases With Dr. Sony Sherpa - Gilmore Health News - September 6th, 2020
- Our teenage son is turning to stone and is becoming entombed in his own body - The Sun - August 28th, 2020
- Gene Therapy: IT Meets Medicine, But Who Is In Charge? - Walter Bradley Center for Natural and Artificial Intelligence - August 24th, 2020
- Edited Transcript of ISEE.OQ earnings conference call or presentation 5-Aug-20 12:00pm GMT - Yahoo Finance - August 6th, 2020
- Opinion: With any COVID-19 vaccine, humanity will face its biggest moral test - The Province - August 4th, 2020
- These Companies Are Seeking a Cure for Sickle Cell. And Its Just the Beginning for Some Gene Therapy Stocks. - Barron's - July 31st, 2020
- FDA Approves Combination Therapy for Treating Advanced Melanoma - Pharmacy Times - July 31st, 2020
- How Can Technology Help Fight the COVID-19 Pandemic? - IoT For All - July 31st, 2020
- Thomas Jefferson University doctor to be honored at W&J commencement - Observer-Reporter - July 30th, 2020
- First Edition: July 27, 2020 - Kaiser Health News - July 30th, 2020
- These are the most valued tech startups in France in 2020 - Silicon Canals - July 7th, 2020
- What's a life worth in dollars and cents? Should that influence who gets treated for expensive disease treatments? - Genetic Literacy Project - July 1st, 2020