The Avanços no Tratamento do Nanismo seminar, held by Folha on November 18, 2025, highlighted scientific advances but pointed to barriers like bureaucracy and prejudice limiting treatment access. Experts and activists discussed the need for public policies and greater awareness. The event, sponsored by BioMarin, featured panels on innovative treatments and inclusion.
The Avanços no Tratamento do Nanismo seminar, promoted by Folha on November 18, 2025 and sponsored by BioMarin laboratory, brought together experts, activists, and parliamentarians to discuss challenges faced by people with this condition. Mediated by reporter Jairo Marques, the 'Innovative Treatments' panel addressed structural barriers to scientific innovation in Brazil.
Pediatric endocrinologist Paulo Solberg from Uerj emphasized delays in research approvals and lack of funding. 'Approving a research project is very time-consuming. And there's the idea that if a First World company wants to do research in the Third World, it's because it wants to take advantage,' he said. Geneticist Wagner Baratela from Hospital Sírio-Libanês and Fleury, who has skeletal dysplasia, advocated private partnerships: 'That classic model of government money helps a lot, but we need more.'
Estimates indicate 3.2 in every 10,000 Brazilians have dwarfism, totaling 65,000 people, with 3 million worldwide. In achondroplasia, the main cause of disproportionate dwarfism, comorbidities like stenosis, apnea, and chronic pain are the biggest challenges, according to Solberg.
Kênia Rio, president of Associação Nanismo Brasil (Annabra), reported ignorance about these comorbidities, affecting the incorporation of vosoritide—the first achondroplasia drug, launched in 2021—into SUS. The request was sent to Conitec in September 2025, but current access requires court actions. 'We are running a campaign to show it's not just a few centimeters,' Rio stated, whose family is in the fourth generation with the condition and whose 12-year-old grandson improved with treatment.
Deputy Rosangela Moro (União Brasil-SP) highlighted that 78% of mothers of children with rare diseases dedicate 100% of their time to care, and advocated laws like 15.120/2025, including civil representatives in Conitec, and the new Clinical Research Law (14.874/2024). 'It's cheaper to treat early so the person can have a normal life,' she said.
In the 'Inclusion Challenge' panel, INN psychologist Izabela Ganzer described daily stress: 'It's like we're in a race that precedes the journey.' Priscila Dornellas, mother of an 8-year-old with achondroplasia, reported school obstacles and invasive approaches. INN's Juliana Yamin demanded recognition of ableism as a collective crime, similar to racism.
Fiocruz geneticist Juan Llerena Júnior noted vosoritide increased visibility, driven by associations and influencers. Despite advances, Rio warned of social invisibility: 'We live in a society that doesn't want to see us.'