Picture a child wanting to move, to play, to simply stand up, and their body quietly refusing, little by little, day after day.
This is the reality for 120+ kids facing Spastic Paraplegia: a progressive, relentless foe written into the GENE.
For Bharat and his son, Devraj, the battle began early. At just 1 year old, Devraj was diagnosed with SPG47. 5 letters that rewired everything. Before he could walk, nerve cells began weakening, muscles began stiffening, leading to delays and terrifying moments when he would gasp for air.
The Silent Epidemic
Devraj is not alone. Today, 120+ children are fighting this exact disorder. They are the reason for late-night medical journal readings and the reason parents learn to fear the sound of a seizure alarm.
The disorder makes walking difficult, makes speech a colossal effort (Devraj has fought for 25 words), and even swallowing and breathing a struggle.
The Call for a Cure
This is where Cure AP-4 comes in; they support families like the Jain family (parents of Devraj) and others diagnosed with SPG47 and other 4 related AP-4 disorders.
This is why Bharat Jain is stepping up with his non-profit fundraising efforts in India.
While the story began with SPG47, the mission extends to finding a cure for the same. The research breakthroughs needed for one of these rare progressive illnesses often light the path for all.
The Science is Ready
The development of this gene therapy has received US FDA clearance to begin a Phase 1/2 clinical trial for children with SPG47.
BlackfinBio is the company that is making the drug for the Phase 1/2 trial.
Families will travel to Boston Children’s Hospital (USA) so their child can undergo the trials for this gene therapy.
The Goal
To actually carry out these trials and safely deliver the treatment through neurosurgery, a fundraising goal of ₹50Crore (5 million US dollars) has been set to cover critical neurosurgery to deliver the gene therapy, hospitalisation, long-term follow-up visits, biostatistics, insurance, and other essential clinical expenses
To give these children their futures back. To move beyond wheelchairs. To enable them to feed themselves, speak their needs, and exist without gasping for breath.
Your donation brings 120+ kids closer to a cure. It goes directly to the gene therapy program, funding the science that could do wonders against these cruel progressive diseases.
Time is running out for Devraj, who has only 20 months left to participate in that trial, and the trial needs to start as soon as possible for him.
120+ families are waiting. Donate!
You are donating to a rare disease to find a cure.
Jain family is targeting to raise 1 million dollars out of the total 5 million dollars needed, as other families all over the world have started working for the same cause.
The goal amount of the campaign may be higher than the attached estimates to address and aid the post-hospitalization expenses/contingencies including but not limited to prolonged medication, diagnostics, rehabilitation therapies, and follow-up doctor visits/consultations which vary from disease to disease.