Research, partnerships, merit, excellence. All our official positions.
What is a genetic disease?
What is a rare disease?
Why are rare genetic diseases known as “orphan diseases”?
Why does Telethon focus on genetic diseases?
How far has research come for the disease I suffer from?
Why isn’t the disease I suffer from in the list of diseases studied by Telethon?
What kinds of research does Telethon finance?
Does Telethon receive any government grants?
How is donors’ money spent?
Who and what are provincial coordinators?
How does Telethon select its partner companies?
When does Telethon grant its patronage?
Does patronage automatically authorise use of the Telethon logo?
Funding research: other official positions
Animal testing is a widely debated and often controversial issue. The main arguments for and against animal trials are outlined on our website.
Telethon pursues cooperative strategies to combine its resources and capabilities with those of private undertakings in order to achieve full therapeutic development of its research results. Read more
The brain drain problem
Working abroad is not only important, it can also be advantageous for researchers, since it broadens their horizons as a part of their all-round training. The problem arises when the flow is one-way, when researchers leave because of the lack of opportunities and career prospects at home. Furthermore, countries with few opportunities will not attract foreign researchers. This has negative repercussions not only for the individuals concerned (when, having considered all the alternatives, they have no option but to leave their own country), but also for the country as a whole (inevitable loss of human and other resources). It is worth noting that this loss regards people who have benefitted from state education, sustained by taxpayers. As far as our activities are concerned, we have attempted to tackle the problem through our careers programme, which is named after the Nobel Prize winner, Renato Dulbecco. This programme enables the best young researchers to set up their own independent research groups in Italy. Read more about the careers programme.
Trials for new therapies
Developing therapies is an extremely time-consuming and complex process, involving many players, requiring considerable economic and other resources (time, expertise, people). Only after it has successfully gone through a number of vitally important stages of development will the therapy truly become a therapy. By completing these stages, we can demonstrate that the therapy is safe and effective. The therapy must have a sound scientific rationale, or, in other words, it must be based on robust, published, replicable laboratory data that have been shared with the rest of the scientific community. The delicate stage of the passage from the laboratory to clinical trials is closely monitored by the regulatory authorities (in Italy these are the Italian Medicines Agency (AIFA), the National Institute of Health (ISS) and the ethics committee of the hospital hosting the trial). These regulatory bodies must define the shared set of rules and ensure full compliance with these, while also safeguarding the health of patients at all times. The clinical trial protocol must be drawn up and include full details of the procedure, including how many and which patients will be involved, which parameters will be used, firstly to assess the safety of the treatment and secondly its efficiency, how long it will last, how much it will cost. Only then can we establish, as objectively as possible, whether a given treatment is of therapeutic value and worthy of being developed and placed on the market for all patients requiring it. Telethon’s view is that any breach of such rules will be harmful first and foremost to the patient. This includes using the term ‘therapy’ to refer to treatments that have been developed in a manner not conforming to the rules shared by the scientific community, and administered in the absence of adequate assessments as described above. We wish to stress that the ‘compassionate’ use of therapies for which no clinical trials have been conducted is also governed by law. Indeed, a treatment that has not been fully tested, or one that is generally administered for other indications, may only be administered to patients suffering from a serious disease for which no therapeutic alternatives are available (as is frequently the case for rare genetic diseases), if the risk-benefit balance is considered to be favourable, and in any case only with the approval of the competent authorities.
Prevention of infectious diseases is one of the key public health objectives. Vaccines have been a tremendous breakthrough in the history of medical practice. Vaccines have all but defeated diseases like smallpox, tetanus, polio and diphtheria. Vaccines have also greatly curbed the incidence of hepatitis B, measles, rubella, parotitis and meningitis. It is paradoxical that vaccines should become the ‘victim of their own success’. Since these diseases have been defeated, or significantly reduced, they are no longer considered to be of great importance. In the meantime, the headlines have been full of alarming stories about the use of vaccines. While scientifically unfounded, these stories have convinced many parents not to vaccinate their children. As a Foundation whose aim is to foster biomedical research into treatments for muscular dystrophy and other rare genetic diseases, we strongly distance ourselves from anyone who uses information that is not based on scientific evidence to persuade people to refuse vaccines. As with all other drugs, vaccines can have adverse side effects. However, current scientific findings indicate that the risks associated with vaccination are significantly lower than the benefits. In any case, the risks are constantly monitored at international level in order to ensure higher and higher levels of safety. It is especially important for patients suffering from rare genetic diseases – the patients we originally set out to assist, and for whom we work every day – to be protected against infections, which could even be life-threatening for people whose health is already fragile. It is also important to note that, by ensuring adequate vaccine cover in the general population, we are also protecting those among us who suffer from diseases that have weakened their immune system to such an extent that vaccination must be ruled out. For further information, please visit the VaccinarSì site, managed by the Italian Society of Hygiene (SITI), the National Institute of Health (ISS), and the Ministry of Health.