About us
We are a nonprofit association from Romania supporting research into genetic therapies for SPG4, with the children living with this condition and their families in mind. This page explains why we exist, what we aim to do and the rules we work by — so that you can hold us to them.
Mission and vision
Mission
To help accelerate research into safe and effective genetic therapies for SPG4, in order to improve the lives of affected children — by funding independently reviewed research, supporting families with verified information and publicly reporting every amount received and spent.
Vision
A future in which a child diagnosed with SPG4 has real treatment options — developed through verifiable science and made available through appropriate medical pathways.
Our values
The principles that guide our decisions, including when it would be easier to do otherwise.
- Scientific rigour
- We support projects with clear hypotheses and sound methods, reviewed by specialists. We say openly what is known, what is not yet known, and which sources we rely on.
- Transparency
- We publish the amounts received, committed and spent separately. Every project we support will have a documented budget, made public before any money is transferred.
- Dignity
- We speak about children and adults with SPG4 as people, not cases. We never use children's stories or images without their parents' explicit consent, and we do not exploit suffering to raise funds.
- Collaboration
- We work with researchers, clinicians, families and patient organisations in Romania and abroad, so that resources are not duplicated and results are shared.
- Independence
- Donors and sponsors do not decide which projects are funded. Selection is based on scientific criteria, and conflicts of interest are declared and managed.
Why this association exists
For parents who learn that their child has SPG4, one of the hardest questions is “what happens next?”. Research into genetic therapies is moving forward in laboratories in several countries, but for a rare disease its pace depends heavily on funding.
Rare diseases seldom attract enough commercial investment. The early stages — cell models, preclinical studies, validating therapeutic targets — are often the hardest to fund, even though their results are what can open the way to public grants and larger studies.
The association started from a question many families of children with SPG4 ask themselves: what can we do, now? The answer was to organise — as a nonprofit with written rules that raises funds responsibly, directs them to independently reviewed research and reports every step publicly.
We do not promise a treatment and we do not give medical advice: genetic therapies for SPG4 are still at the research stage. Our aim is for every amount donated to go where it can matter most for science and for children — and to be able to show, at any time, where it went.
The founders’ story will be published here, with their consent.
What we aim to do
Our concrete objectives. We will report progress on each of them publicly, including when things move more slowly than we would like.
Fund reviewed research
Support projects working towards genetic treatments for SPG4, selected through independent scientific review, with documented objectives, budgets and timelines.
Build an SPG4 community in Romania
Bring together children and adults with SPG4 and their families and, subject to ethics committee approval, start a patient registry that can support future studies.
Inform families accurately
Explain the condition and the state of research to parents in Romanian, clearly and with verified sources, without unfounded promises.
Connect with international research networks
Work with research groups and patient organisations abroad, so that families in Romania have access to information and, when the time comes, to clinical studies.
Report publicly
Regularly publish the state of our funds, our funding decisions and the results of the projects we support.
Leadership and team
The members of the board and the team will be listed here once each person has confirmed their role and agreed to be named publicly.
Scientific advisory board
Specialists who independently review research proposals and help us understand the state of the science.
Darius Ebrahimi-Fakhari
Boston Children's Hospital · Harvard Medical School
Darius Ebrahimi-Fakhari, MD, PhD, is Director of the Movement Disorders Program at Boston Children's Hospital and Assistant Professor of Neurology at Harvard Medical School. His laboratory studies genetic and molecular causes of childhood-onset movement disorders and neurodegenerative diseases. His hereditary spastic paraplegia research includes natural history studies of early-onset forms, including SPG4, and gene therapy work for AP-4-associated HSP.
Miguel Sena-Esteves
UMass Chan Medical School
Miguel Sena-Esteves, PhD, is Associate Professor in Genetic and Cellular Medicine at UMass Chan Medical School and leads its Translational Institute for Molecular Therapeutics. His laboratory develops AAV gene therapies for neurodegenerative lysosomal storage diseases, including Tay-Sachs disease. He was co-senior author of a study in which SPAST-AAV9 gene therapy, given to newborn mice, prevented symptoms in an SPG4 model.
Peter Baas
Drexel University College of Medicine
Peter Baas, PhD, is a Professor of Neurobiology & Anatomy at Drexel University College of Medicine. His laboratory studies microtubules, part of the neuron's internal skeleton, as a shared target in neurodegeneration, injury and developmental disorders. His team developed an SPG4 mouse model; Baas was co-senior author of a study in which SPAST-AAV9 gene therapy prevented symptoms in these mice.
Liang Oscar Qiang
Drexel University College of Medicine
Liang Oscar Qiang, MD, PhD, is an Associate Professor in the Department of Neurobiology & Anatomy at Drexel University College of Medicine. His laboratory uses human induced pluripotent stem cell (iPSC) models, including cortical organoids, to study tauopathies and hereditary spastic paraplegia. His SPG4 research examines how SPAST mutations damage axons and identified HDAC6 as a potential therapeutic target.
Legal details
The association’s legal details. We add them as they are officially confirmed.
- Registered name
- ASOCIAȚIA IVACATERINA’S WAYMAKERS
- Tax identification code (CIF)
- 50179280
- Number in the Register of Associations and Foundations
- 1441/A/2024
- Registered office
- Str. Dropiei nr. 2, sc. D, et. III, ap. 14, Timișoara, jud. Timiș
- Date of establishment
- 4 June 2024
- ANAF register of entities eligible for tax deductions (RUC)
- to be confirmedRUC registration is the condition for companies to subtract sponsorship from their profit tax and for individuals to redirect 3.5% of their income tax on salaries.
- Data protection contact
- privacy@ivacaterina.org
- Statute of the association
- to be confirmed
Annual reports
Activity reports and financial statements, published for anyone who wants to check them.
The first annual report will be published after the end of the first financial period. Until then, verified amounts received are updated on the transparency page.
Go to the transparency pageGovernance and commitments
The rules we commit to publicly. You can hold us to account for each of them.
Conflict-of-interest declarations
We commit that board members and reviewers declare in writing any link with the institutions, companies or people involved in a decision, and do not take part in decisions in which they have an interest.
Independent scientific review
We commit that every research proposal is reviewed by specialists independent of both the association and the proposing team before any funding decision.
Grants approved by the board
We commit not to award any amount to a project without a written board decision, based on the scientific review and a documented budget.
An auditable record of financial changes
We commit to keeping a log of every change to our financial records — who made it, when and why. Incorrect entries are never deleted, only voided with a stated reason.
Pledges kept separate from money received
We commit to reporting pledged, actually received, contractually committed and spent amounts separately. Only money received and verified counts as funds raised.