Theorem Clinical Research

SMA Foundation

Spinal muscular atrophy program enters phase Ib/IIa trial in patients with SMA

Thursday, November 20, 2014 01:19 PM

PTC Therapeutics’ joint development program in spinal muscular atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a phase Ib/IIa study in adult and pediatric patients. The placebo-controlled, randomized, multiple-dose study will enroll approximately 48 patients with SMA and investigate the safety and tolerability of an investigational survival of motor neuron 2 (SMN2) gene splicing modifier (RG7800) over 12 weeks. The achievement of this milestone triggers a $10 million payment to PTC from Roche.

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Quest Diagnostics

PTC Therapeutics, the SMA Foundation and Roche collaborate

Friday, January 24, 2014 10:39 AM

PTC Therapeutics, the SMA Foundation and Roche have 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. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the leading genetic cause of death in infants and toddlers.

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PTC Therapeutics announces milestone in SMA collaboration

Tuesday, August 13, 2013 10:00 AM

PTC Therapeutics has announced the selection of a development candidate in its spinal muscular atrophy (SMA) collaboration with Roche and the SMA Foundation. The achievement of the milestone triggers a $10 million payment to PTC from Roche.

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Roche, PTC sign potential $490m licensing deal

Tuesday, November 29, 2011 01:56 PM

Roche, PTC Therapeutics, and the SMA Foundation, have signed a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) program. SMA is a genetic neuromuscular disorder that causes muscle weakness.

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CWWeekly

March 30

Icon, Carnegie Mellon partner to research ways to improve study patients' understanding of Informed Consent

Veeva launches OpenKey, partner program to unlock difficult access to, restrictions on customer reference data

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The CenterWatch Monthly

March

Novo Nordisk, Takeda, Servier, Celgene among top sponsors
New gains seen from patient- and site-centric initiatives

Patient reported outcomes take center stage
FDA combining PROs, clinical outcomes in its approval decisions

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The CenterWatch Monthly

February

Study grants market breaks $13 billion
Volume of clinical activity rising while growth in grant spending slows

Revisiting eClinical Technology Solutions Adoption
While use of established solutions grows, sponsors hesitate on newer tools

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