Tuesday, November 15, 2022

Treating achondroplasia: Ascendis releases outcomes of the phase 2 study with TransCon-CNP

The ACcomplisH trial

 On November 13th, Ascendis Pharma released top line results of the ongoing phase 2 study with TransCon-CNP, an analog of c-type natriuretic peptide (CNP) wrapped in a transport molecule to allow slow-release and extend the half-life of the analog. 

Let's pause here for a moment as in this first sentence there is a lot of information already. 

In other words, TransCon-CNP is a competitor of vosoritide. The main difference between them is that the CNP analog from Ascendis uses a kind of taxi: its CNP analog is covered by a molecule that serves as a protection against the body clearance systems, so it has an extended time to exert its actions. The taxi allows the Ascendis CNP analog to be given just once a week as opposed to vosoritide, which needs to be administered daily.

As typical of phase 2 studies, the ACcomplisH trial was designed to evaluate several different doses to TransCon-CNP in order to define which one would have the best safety/efficacy profile.

In summary, these are the headlines directly from Ascendis press release

  • In the Phase 2 ACcomplisH Trial in children with achondroplasia aged 2-10, once-weekly TransCon CNP demonstrated the potential to meet patient and caregiver needs for a safe, effective, tolerable, and convenient treatment
  • The primary endpoint, annualized height velocity (AHV) at Week 52, demonstrated superiority of TransCon CNP at 100 μg/kg/week compared to placebo (p=0.0218)
  • TransCon CNP was generally well tolerated with low frequency of injection site reactions; all 57 randomized children continued, with the longest treatment duration beyond two years
  • Data showed robust and consistent results in prespecified analyses across age groups and dose levels, supporting continued development at the selected dose of 100 μg/kg/week


Three of the four doses tested lead to growth improvement but only the highest one was found to be significantly superior to placebo (Table 1).

It is important to learn that TransCon-CNP worked similarly in all age groups tested.

Table 1. Efficacy of TransCon-CNP weekly doses (from the AComplisH study presentation).



The press release informs us that TransCon-CNP was well tolerated. Most of the few adverse events reported were related to local injection site reactions.

Next steps

Based on the results of this study Ascendis informed that they are in conversations with regulators and also enrolling patients in their phase 2B study to further investigate the chosen dose for achondroplasia (100 µg/kg/week).


As we can see in Table 1, children  the highest dose of TransCon-CNP on average grew 1.07cm (24.6%) more than those in placebo. This is lower than what was obtained with vosoritide in its phase 3 study (1,2), which you can see below (transcript from the original publication;(2)):

  • In the placebo-controlled study, children randomized to treatment with vosoritide increased annualized growth velocity to 5.96 (1.51) cm/year at 26 weeks and 5.39 (1.87) cm/year at 52 weeks. In children randomized to placebo the annualized growth velocity was 4.08 (1.36) cm/year at 26 weeks and 3.81 (1.31) cm/year at 52 weeks.

Based on what we have already seen with vosoritide trials, one aspect to have in consideration is that there is a large variability of response to treatment (just look at the growth ranges among the treated groups in Table 1). This was also seen in vosoritide studies. 

The population treated in the phase 2 study with TransCon-CNP is considerably smaller than the one in the phase 3 vosoritide trial, but the results show the potential efficacy of the chosen dose that we would expect in the ongoing phase 2B study. It would be interesting to learn why the developer did not consider evaluating a higher dose of TransCon-CNP, as it is possible that its optimal dose (efficacy+safety) might not have been identified yet.

One relevant advantage of TransCon-CNP is its weekly dosing schedule, which would be likely considered more comfortable by parents and individuals than vosoritide's daily injection. 

Finally, if TransCon-CNP would be able to provide consistent growth in longer term (as we might see in the next step of its clinical development), and taking in account that it has been showing to work positively in younger kids, it might become a fair option to vosoritide in the future, even if the nominal growth increment was lower in the phase 2 study compared to the already approved treatment.


 1. Savarirayan R et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet 2020; Oct 10;396(10257):1070. Free access.

2.  Savarirayan R et al. Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genet Med 2021 Dec;23(12):2443-47. Free access.

Thursday, October 20, 2022

Treating achondroplasia: where do we go from here?

Just a few days ago, Fundación Alpe Acondroplasia was inaugurating its seventh international congress about skeletal dysplasias (http://www.congresoalpe2022.com/). Top experts from all over the world came together to present current best practices in the healthcare for achondroplasia and skeletal dysplasias and talk about the advances seen in the last decade. 
Drug developers provided updates about their current work. Most importantly, the congress brought to Gijón, the charming Asturian town home of Alpe (Figure 1), leaders from many parts of the globe to discuss the main challenges those with skeletal dysplasias face in a daily basis, from social discrimination and political issues to access to appropriate healthcare in their countries.
Figure 1. Gijón, Spain.
Gijón, Spain.


Fundación Alpe has been a stronghold for skeletal dysplasias throughout the years, with a clear vision of what is needed today to improve the lives of people with skeletal dysplasias but also with an understanding that, as science evolves, what is considered standard now may become obsolete in the future. I believe that vision explains why they set Alpe y un nuevo horizonte (Alpe and a new horizon) as the signature for this edition of the congress.

As a member of Alpe's Scientific Advisory Board, I was asked to talk about the changing scenery in the healthcare for achondroplasia and skeletal dysplasias. I am grateful to the strong leaders of Fundación Alpe, Carmen Alonso Alvarez and Susana Noval Iruretagoyena for the opportunity to talk about the current status of pharmacological therapies for achondroplasia and future perspectives during the congress. 

Here I share my presentation with our 17 readers, hoping that the topics discussed there (and here) will help families interested in therapies for skeletal dysplasias to stay updated about the developments we are seeing in the field.

So, where do we go from here?

Achondroplasia has been known for thousand of years. Archeological treasures have been found in all continents depicting the typical features of this skeletal dysplasias (Slide 1).

 Slide 1. Achondroplasia has been depicted by many ancient cultures all over the world.

 A long journey

The term achondroplasia was used for the first time in 1878 by the French doctor Jean Parrot to describe a condition where there was no cartilage growth (Slide 2).

It took more than a hundred years from that first paper for science to identify the gene mutation that causes achondroplasia. A recurrent, single point mutation in the gene which encodes the enzyme Fibroblast Growth Factor Receptor 3 (FGFR3) was finally linked to achondroplasia in 1994. After that, scientists quickly learned that the recurrent G380R mutation in FGFR3 makes this receptor enzyme excessively active leading to bone growth arrest.

Meanwhile, a few pioneers started to study the mutation and almost ten years later the first attempts to find therapies were published. In 2003, Dr. Yayon group designed the first antibody against FGFR3 while the Japanese group leaded by Dr. Nakao released the first study with C-type natriuretic peptide (CNP) in the cartilage growth plate. CNP was discovered to antagonize the activity of FGFR3 in the growth plate and this lead to further discoveries.

In 2010, Biomarin introduced a first CNP analog, BMN-111, in their pipeline. In 2014 they started the first study of BMN-111 (now known as vosoritide) in children with achondroplasia. Finally, in 2021, after a long clinical development journey, vosoritide was approved by major regulatory agencies and is already being used in several countries for the treatment of achondroplasia.

Slide 2. A timeline of achondroplasia.

One aspect of the scientific research about achondroplasia is that the interest about this skeletal dysplasia got a booster after the announcement of the phase 2 study of vosoritide (Slide 3). Someone reviewing the scientific activity about achondroplasia might reach the conclusion that the increased interest was mostly driven by drug developers introducing new potential therapies. This is only partially true. In fact, although it has been recognized that achondroplasia is the most common form of skeletal dysplasia, and that a relatively fair knowledge about its typical clinical features were already in place in 2004, many questions regarding clinical and developmental aspects were still poorly addressed. In the last few years many publications have focused on epidemiological, clinical and developmental aspects of achondroplasia rather than simply working with potential therapies (Slides 4 and 5).

Slide 3. The number of publications about achondroplasia has been rising in the last ten years.


Slide 4. Research on drug therapies for achondroplasia leads to improvement in healthcare.


Slide 5. The first International Consensus for the management of achondroplasia.


Real people. Real problems.

Why are these new guidelines and a first international consensus on healthcare for achondroplasia so important? 

In the real life, daily medical and social challenges are common place both for children and adults with achondroplasia (Slide 6 just provides a few examples extracted from social media). These new publications aim to address the key medical elements that may improve health and quality of life spanning all age groups. It is clear that we need to do better.

Slide 6. Real people. Real problems.


While learning and understanding the natural history of achondroplasia will certainly lead to better healthcare, it is undeniable that the research towards solutions to control the overactive FGFR3 are in full speed right now, with many potential therapies in various degrees of development (Slide 7).

 Slide 7. Current and potential therapies for achondroplasia.

As we know, last year vosoritide was approved for the treatment of achondroplasia based on its positive effects on bone growth velocity and incremental and sustained growth over two years (Slide 8). There was some controversy about choosing growth velocity as a parameter of efficacy without observing other clinical aspects that are relevant in achondroplasia. However, having in mind that bone growth is a slow process, using changes in growth velocity seems to be a reasonable way to verify efficacy in relatively short term. The known clinical complications seen in achondroplasia are frequently related to restricted bone growth. It is possible that over years (so long term) we may see that these complications will become less prevalent, but this will take some time more to be confirmed.

Furthermore, if together with improvement in standing height, we expect that there will be a reduction in the frequency of medical issues in children receiving pharmacological therapy, then another aspect that should also improve is quality-of-life (as measured through several tools), which is known to be lower in achondroplasia compared to the general population.

Another important question that needs to be addressed has to do with an understandable but unrealistic expectation many parents may have today about the results of any pharmacological therapy (Slide 8). Having their child under drug treatment should not preclude the need for continuous specialized follow up, at least for the next few years, until we get more evidence that the treatment may really reduce the incidence of orthopedic and neurological complications. Let's say we need to wait and see whether the treatment could bring the child to a level of healthcare need that is typical of the average child. So, as for now, just pharmacological treatment is not enough.


Slide 8. The changing landscape.

So, where are we now and where do we go from here? (Slide 9)

As countries approve and include vosoritide within their pharmacopeias, there have been many questions raised on social media related to access to treatment. Vosoritide is a high cost medicine, at a level that is not affordable for the vast majority of families interested in offering this option to their children. The developer and payers must find a common ground where the reward for being the pioneer in the field is well paid off while access is amplified. 

However, access is only one the challenges ahead. The social media is indeed a source of helpful information (at least related to our topic here). An issue that has been raised by families looking for treatment for their kids is the little, if any, knowledge professionals that assist kids with achondroplasia have been demonstrating about the new pharmacological therapy and healthcare management in general. Since experts in skeletal dysplasias usually work in specialized referral centers, effort needs to be done to spread more effectively the knowledge not only about pharmacological therapies but also on the management of achondroplasia in the primary or secondary care settings. This, in turn, may also help improving access to therapies. Statement papers and the Consensus depicted in slides 4 and 5 may be a good starting point to extend the knowledge gained in recent years to the front-line of healthcare.

Slide 9. The future of healthcare for skeletal dysplasias.


A new horizon

Finally, as Alpe has envisioned in the recent conference, a new horizon is ahead. With all the research performed to understand the FGFR3 mutation in achondroplasia, how it works and affects bone growth, much has been learned about the overall bone growth and development process. 

The FGFR3 pathway is a powerful growth brake in the cartilage growth plate. It is a very important modulator of bone growth working to balance the activity of several other agents that promote bone growth. If it is working less than normal bones grow excessively and may cause medical issues; the same occurs when it is working too much as we see in achondroplasia.

We now know that FGFR3 activity can be controlled. The question is whether this knowledge can be used to see if controlling FGFR3 activity would be helpful for other skeletal dysplasias. The answer to this question is yes (Slide 10). 

It would be common sense to deduct that other skeletal dysplasias where in some way the FGFR3 pathway is unbalanced could be managed by vosoritide or other drugs in test for achondroplasia. For instance, other FGFR3-related dysplasias such as hypochondroplasia or other very rare cases where FGFR3 mutations cause proportionate short stature. 

Another example would be the CNP-related dysplasias. In these cases, most of the time the mutation causing the dysplasia is located in the CNP receptor, so giving CNP analogs would not likely help improving bone growth, but agents targeting FGFR3 directly, such as infigratinib, could be helpful.

Another example shown in Slide 10 are the RASopathies, such as Noonan syndrome, where the same enzymes (the MAPK pathway) activated by FGFR3 are overactive due to mutations in regulatory proteins. In this case inhibiting FGFR3 could help reducing MAPK activity to improve bone growth.

Other skeletal dysplasias might benefit of the use of anti-FGFR3 agents and there is already evidence in the literature, for instance in diastrophic dysplasia and in Mckusick syndrome,  that FGFR3 might be overactive and contributing to impaired bone growth.

It is also foreseeable that treatments developed for achondroplasia might benefit children born with other bone growth impairment conditions. It is time to multiply the efforts to evaluate these new potential therapies for other skeletal dysplasias.

 Slide 10. Treating other skeletal dysplasias



The future is around the corner

Skeletal dysplasias are disorders which affect the body in development. These pharmacological therapies may, in the near future, help children to grow better, bringing together many benefits that go beyond achieving a higher final stature. One of them, which I have already referred to in the past, is very simple. Let's just imagine a time where a child with achondroplasia will be living the same ups and downs any average child lives as they grow into adulthood. No more, no less. I think this is a good perspective to have.

Saturday, February 12, 2022

Treating Achondroplasia: ten years online. A review of the current achondroplasia therapy landscape

Ten years in a row

The blog Treating achondroplasia is celebrating 10 years online. I have not been posting as often as some years ago but I keep my eyes open to all new relevant information that comes to the field and share them with the interested reader here. 

Ten years ago, I started this blog with the main objective of translating the hard jargon that is typical in scientifc publications into a more relatable language that could be accessible to all. The blog has received more than 430K visits since its launch and I hope it has been a helpful source of information for you.

So, to start this new year (I know, it's already February) I want to share with you an updated review about therapies for achondroplasia that I prepared for Fundación Alpe (Gijón, Spain). Alpe is one of the world's strongest advocacy groups for acondroplasia and skeletal dysplasias and you can find the original article translated to Spanish here. This review provides you with high level information about all drugs that are known to be (or could be) in clinical development for the treatment of achondroplasia. You can find more details about them here in the blog: you just need to search for them in the index page.

Nevertheless, the research for therapies does not stop with the drugs listed in Table 1 below. New therapeutic approaches are being explored that we will be reviewing in the next blog's article.

Thank you for your interest in the Treating Achondroplasia blog!

An update of the therapies for achondroplasia

Note: this review has been prepared originally for Fundación Alpe.

Achondroplasia is the most common form of short-limbed dwarfism. This skeletal dysplasia is caused by a single point mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, which in turn encodes the protein FGFR3, which is located across the cell membrane of the chondrocytes (1). Upon binding of its ligands (the FGFs), it is activated and drives several important cell functions (Figure 1).

FGFR3 has a key role in bone development by regulating the growth plate cartilage function. FGFR3 helps regulating bone growth by, like a brake in a car reducing its speed, counteracting the effect of many other agents which, as the car accelerator, promote bone growth (1). Without FGFR3, bones would elongate without control causing several medical complications (2). In achondroplasia, however, because of the mutation, FGFR3 is working a little too much thereby impairing bone growth (1). More potent mutations in FGFR3 lead to significantly more severe, sometimes lethal forms of skeletal dysplasia.

The consequences of the FGFR3 mutation are well known and go beyond the short stature typical of achondroplasia. Sudden death and neurological problems in early infancy, sleep apnea, recurrent ear infections and multiple orthopedic complications among others throughout life, not to mention significant impact on the quality-of-life, have been already extensively documented in many studies (3,4).

Since the mutation was discovered much has been learned about how it causes achondroplasia. Scientists started thinking and investigating how to control or reduce the activity of FGFR3 to help restoring bone growth. For instance, one of the first objective attempts to target FGFR3 for the treatment of achondroplasia was explored by the group led by Dr. Avner Yayon, who developed an antibody that could block FGFR3 and its activation (5). Unfortunately, FGFR3 works, as we saw above, in the growth plate cartilage, a very special tissue located within the very ends of each of our bones. The growth plate cartilage is a dense tissue that does not receive direct blood supply and these singular features prevent large molecules to transit inside it. As antibodies are very large molecules, in contrast to their vast use in treating many other diseases and in particular cancer, they couldn’t reach their target (FGFR3) in the growth plate, making them inappropriate for the treatment of achondroplasia.

Nevertheless, as scientists were mapping the chemical reactions driven by or affecting FGFR3 (Figure 1), they learned about many other bone growth-promoting agents, too. For instance, they learned that the C-type natriuretic peptide (CNP) pathway plays a key role in bone growth and that it also antagonizes FGFR3 in growth plate chondrocytes. Increasing CNP levels in the growth plate restores, at least partially, bone growth (6,7). With this knowledge in hands, vosoritide has been developed (8), followed by TransCon-CNP (9).

Figure 1. Pharmacological strategies targeting the FGFR3 pathway in the chondrocytes.


Modified from Matsushita M et al. 2013 (10). Reproduced here for educational purposes only.

As FGFR3 may play important roles in some types of cancer, scientists have tried to block it with molecules called tyrosine kinase inhibitors (TKIs) which have the ability to “turn it off” or deactivate it. It was natural to think that a TKI could be explored in achondroplasia. Actually, two of them are in development for achondroplasia (see below) and others might follow the clinical development path (Figure 1).

Scientists also paid attention in how FGFR3 is activated and if it was possible to prevent it. This resulted in the design of molecules like recifercept, a modified FGFR3 molecule that can circulate free, capturing the activators (FGFs) before they reach out to the genuine FGFR3. Using the same strategy, another molecule called aptamer was designed to do the same job, blocking one of the key FGFs before they turn on FGFR3 (Figure 1).

You can find a list of the pharmacological agents being explored for the treatment of achondroplasia on Table 1 and more details about them below.

Table 1. List of current and potential therapies for achondroplasia.








CNP analog






CNP analog




Phase 2






Phase 2


Ligand trap




Phase 2



Nagoya University



Phase 1


FGF2 Aptamer




Phase 1






Phase 1








CNP analog





CNP: C-type natriuretic peptide. RoA: route of administration. SC: subcutaneous. TKI: 


After long years of clinical development culminating with a successful phase 3 study (11), vosoritide, branded as Voxzogo, has been approved for the treatment of achondroplasia in 2021. In Europe (EMA countries) and Brazil, vosoritide has been approved for children two years of age and older while, in the US, the Food and Drug Administration (FDA) authorized the treatment for children from five years old onward. Other countries will soon follow suit.

Beyond the phase 2 and 3 studies in older children, Biomarin is also testing vosoritide in other three clinical trials, one in infants (NCT03583697), one in children with achondroplasia at higher risk of clinical complications (NCT04554940) and also in a study with other selected forms of genetic growth disorders (NCT04219007). 


The main difference between TransCon-CNP and vosoritide is that TransCon-CNP is delivered through a slow-release system allowing a weekly dose with sustained exposure to their analog in contrast with vosoritide's daily dosing. In pre-clinical studies they showed that their CNP analog was superior to vosoritide (9).

Ascendis Pharma is conducting the phase 2 study ACcomplisH with TransCon-CNP. During the JPMorgan 2022 Healthcare Conference in early January (Figure 2), they reported that TransCon-CNP has been well tolerated during the study, with already 65 weeks of drug exposure. They plan to release the data from the phase 2 study in the end of 2022.

Figure 2. ACcomplisH study design (from Ascendis’ JPMorgan 2022 Healthcare Conference presentation).


Infigratinib is an oral molecule initially developed to treat several types of cancer where FGFRs play an important role for the progression of the disease. It works by blocking the FGFRs' signaling cascades inside the cell (Figure 1). Given that an abnormal, overactive FGFR3 is the cause of achondroplasia, investigators sought to find whether infigratinib could be used to treat this skeletal dysplasia. Preclinical studies demonstrated that it rescued bone growth in a mouse model of achondroplasia, in doses much lower than those used in the first studies in cancer (12,13).

QED, a BridgeBio arm, has been conducting the phase 2 trial called PROPEL and, according to their presentation during the JPMorgan 2022 Healthcare Conference, infigratinib has been showing a safe profile. They estimate to have results from the study by the end of the second quarter this year (Figure 3). Depending on the results they plan to open the phase 3 study right in 2023.

Figure 3. Phase 2 study PROPEL design (from the BridgeBio’s JPMorgan 2022 Healthcare Conference presentation).


Recifercept is a modified, free form of FGFR3 that works as a "ligand trap", capturing FGFR activators (ligands: the FGFs) before they can bind and activate these receptors, including FGFR3. Without the activators FGFR3 would not be as active as expected and this would help restoring bone growth (14,15).

Pfizer has started a phase 2 study with recifercept in the end of 2020 but there have been no significant updates since then. 


Drug repurposing is a strategy where investigators try to find new therapeutic indications for old drugs. The concept is that its development for the new purpose should be much less expensive and the final drug cost, if approved, would be surely more affordable than the cost of newly created compounds. Meclizine is an old drug that has been used to treat motion sickness for decades. In an effort to find potential treatments for achondroplasia the Japanese group from University of Nagoya leaded by Dr. Kitoh has found that meclizine was able to inhibit the FGFR3 function and to partially rescue bone growth in their animal model (10,16). They have subsequently conducted a phase 1 study in children with achondroplasia (17). The study showed that meclizine could be suitable for a single daily dose (but that it would need to be further explored in subsequent studies). More recently, they conducted another study to evaluate multiple doses of meclizine for a period of two weeks, but no results have been published yet.


Ribomic has been developing RBM-007, an anti-FGF2 aptamer designed to treat conditions where FGF2 has a relevant role in the mechanism of disease (18). Since FGF2 is considered a key activator (ligand) of FGFR3 and that in achondroplasia FGFR3 is overactive, then if it was less activated by FGF2 perhaps bone growth could be restored.

Ribomic published their pre-clinical studies with RBM-007, which indeed rescued bone growth in a model of achondroplasia (19). They have already started a phase 1 clinical trial to evaluate this aptamer for achondroplasia and are planning to start a phase 2 study in children with achondroplasia during 2022 (Figure 4).

Figure 4. Clinical development plan of RBM-007 for achondroplasia (from Ribomic’s JPMorgan 2022 Healthcare Conference presentation).


Last year, Sanofi announced that SAR442501, an anti-FGFR3 antibody, was transitioned to Phase 1 clinical development. However, no study information could be found on the European Clinical Trial Register, on ClinicalTrials.gov, or the Australian Clinical Trial Register. Apart from being listed in the Sanofi’s pipeline website and in their Feb 2021 financial report (and other reports in their website), no further information about the development of SAR442501 could be found there. Neither pre-clinical data could be found in the Pubmed portal nor through a Google search as of 31-Jan-2022.

Although the antibody strategy is attractive due to its high specificity, the ability of a specific antibody to target FGFR3 in the growth plate in a model of achondroplasia needs to be confirmed in an appropriate model. As mentioned above, the growth plate is a unique body tissue because it lacks direct blood supply. Nutrients and oxygen must traffic through a dense matrix which involves the chondrocytes. In this setting, large molecules such as antibodies might not be able to reach out to chondrocytes to exert their effects (20).

SAR442501 is not the first antibody against FGFR3 to be developed (6). Until recently, B-701 (R3Mab) (21), also known as votafamab, was being developed for cancer and potentially for achondroplasia, but it seems that the research for this indication has been abandoned as there have been no reports about this antibody for this indication for a long time. 


Astellas Pharma has recently published a study where they explored the use of ASP-5878, a TKI similar to infigratinib, in pre-clinical models to treat achondroplasia (22). They found that the drug was able to improve bone growth, however it was less effective compared to a positive control, a CNP analog bearing the same structure of vosoritide. 


Asubio, a Japanese biotech that was recently incorporated by Daichii-Sankio (DS), was developing another CNP analog based on the fusion of the active fragment of CNP and a backbone fragment of the hormone ghrelin to help extending the known short CNP’s half-life. They have published some studies showing that their molecule was able to improve bone growth in pre-clinical models (23) but there has been no news about this compound in the DS website or in the literature lately.

A new era started

With the approval of vosoritide, a new era started. Treating achondroplasia goes far beyond simply improving the final individual height, which nevertheless is an important objective. Although long term data about the effects of vosoritide (and of course the others, too) is not available yet, based on current evidence, there is a fair chance that these therapies may mitigate or prevent several clinical challenges children with achondroplasia face in their daily routine, as mentioned in the beginning of this review.

These children now may have access to a therapy that might help them develop better and have the same opportunities and challenges an average child has while they grow into adulthood. Neither more nor less. I think this is a good perspective. 


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