A Christmas celebration for children at-risk for HD
December 2025 marked another meaningful journey to Colombia for our annual Christmas celebrations with families affected by Huntington’s disease. Each year, across Peru, Colombia, and Venezuela, our local teams gather children at risk for Huntington’s disease together with their families—not simply for a holiday event, but to affirm community, dignity, and solidarity. These celebrations are […]
Factor-H colabora con Skyhawk para diseminar información sobre el estudio Falcon-HD
Factor-H tiene como prioridad compartir información sobre la investigación clínica de una manera responsable, sin generar expectativas irreales ni aumentar la carga emocional de las familias afectadas por la enfermedad de Huntington. El estudio Falcon-HD está evaluando SKY-0515, un fármaco experimental de administración oral desarrollado por Skyhawk Therapeutics para modular la expresión del gen HTT, […]
Factor-H and Skyhawk Therapeutics collaborate to Support Responsible Communication on the Falcon-HD Clinical Trial in Latin America
A central priority for Factor-H is ensuring that information about clinical research is shared in a way that does not create unrealistic expectations or add to the emotional burden faced by individuals and families affected by Huntington’s disease. While scientific progress is essential and provides reasons for optimism, it is also a long and complex […]
In memory of the Maestro and in honor of his Sister – love and living in ambiguity
On January 3rd, 2026, at 7 am (UK time), my “Hermanito” Gindel rang me. ‘They’re bombing Caracas’ he said, ‘don’t worry, I love you, I’m safe’, ‘I’ll call you later’. A call like that leaves you in a state of anxiety. Fear of the unknown. And I’m not living there. Gindel, like all our Habitat […]
A few words on Skyhawk’s Phase 1c interim results – SKY-0515 in Huntington’s disease
Dr. Ignacio Muñoz-Sanjuan, Factor-H president, shares in this article his first insights on the Skyhawk’s Therapeutics announcement from January 28. There are always two sides with any important development regarding possible treatments for a devastating condition like Huntington’s Disease: one is the scientific part of it; the other is how the results of any trial […]
The girl at the end of the world
A story by Alex Fisher “In the small space that we have built so much on, it’s not just their genes but their everyday which is a moral laboratory, and the girl at the end of the world should remind us of that.” Alex Fisher is an occupational therapist who has worked with HD patients […]
Interview with Ignacio Muñoz-Sanjuán – Reflections on UniQure’s AMT-130 study
Explanation in Spanish of the meaning of UniQure’s new AMT-130 therapy, with clarifications on the what and the when, by Dr. Ignacio Muñoz-Sanjuán (based on the most recent study published on September 24, 2025)
AMT-130 Marks a Historic Moment for Huntington’s—Now Comes Careful Communication
On July 10, 2024, I wrote a reflection piece following the investor conference where uniQure presented the encouraging 24-month results of AMT-130, the first HTT-directed gene therapy delivered directly into the brain regions most severely affected by mutant huntingtin —the caudate and putamen. At that time, early evidence suggested the therapy might exert a stabilizing […]
2025 Dia da Gratidão - um resumo dos eventos na Venezuela
O Dia da Gratidão ocorreu em 23 de março de 2025 e culminou uma semana de eventos na Venezuela para homenagear as contribuições das famílias venezuelanas para o avanço da ciência e da medicina. A Factor-H, trabalhando em conjunto com a Habitat LUZ, voluntários da comunidade e profissionais de saúde, organizou uma série de eventos que variaram de uma atividade médica pediátrica em [...]
Dia da Gratidão 2025
"O Dia da Gratidão é uma forma de homenagear a colaboração das famílias de portadores da DH com médicos e cientistas, que começou com os esforços para identificar o gene da doença de Huntington na década de 1980 e que continua até hoje por meio de vários esforços em todo o mundo para encontrar tratamentos para a doença.
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