PTC Therapeutics, the SMA Foundation, and Roche, announced that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers.
Luca Santarelli, Head of Neuroscience, Ophthalmology and Rare Diseases at Roche commented: "The compelling science behind this project and the highly synergistic alliance among Roche, PTC Therapeutics and the SMA Foundation are the groundwork for this research program. Although still early in development, we consider every step towards a therapeutic option in SMA of high importance for patients and families affected by this devastating and currently untreatable condition."
Luca Santarelli, Head of Neuroscience, Ophthalmology and Rare Diseases at Roche commented: "The compelling science behind this project and the highly synergistic alliance among Roche, PTC Therapeutics and the SMA Foundation are the groundwork for this research program. Although still early in development, we consider every step towards a therapeutic option in SMA of high importance for patients and families affected by this devastating and currently untreatable condition."
"This important achievement may pave the way toward a brighter, more hopeful future for children and adults with SMA," said Loren Eng, President of the SMA Foundation. "While we appreciate that the safety studies in healthy volunteers are an early step and much further development will be required, this is a significant accomplishment, and we are grateful to the commitment of our partners in accelerating this program as rapidly as possible."
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