Dear Esteemed Executives,

We, a collective of patients who have experienced the profound impact of Charcot-Marie-Tooth Type 1A (CMT1A), write to you with a plea for compassion and urgency. Our lives have been profoundly affected by this debilitating condition, and we implore Pharnext to consider providing access to the medication PXT3003 through the FDA Expanded Access Program.

Allow us to share our heartfelt reasons for this request:

Some of us have participated in the clinical trial for PXT3003. During this time, we witnessed firsthand the potential benefits of this medication. It was a beacon of hope, alleviating our pain and enhancing our mobility.

Imagine our devastation when we were informed that the clinical trial participants must abruptly cease taking PXT3003. The sudden withdrawal left us grappling with pain, discomfort, and a loss of hope. PXT3003 was not merely a medication; it was a lifeline. Its efficacy was evident, and its tolerability remarkable. Pain receded, and our mobility improved. We could once again engage in daily activities with greater comfort.

Now, we find ourselves desperate. The pain has returned, and our mobility is compromised. We yearn for the relief that PXT3003 provided. It is not just a medication; it is our lifeline.

We beseech Pharnext to consider allowing PXT3003 to be accessible through the FDA Expanded Access Program. Our lives hang in the balance, and this medication can make an immense difference. We understand the complexities of drug development, but we implore you to see the human faces behind the data.

In unity, we sign this letter, representing the collective voice of CMT1A patients who seek solace, relief, and hope. We trust that Pharnext will recognize the urgency of our plea and take compassionate action.

Thank you for your consideration,

Patients of Hope & Understanding