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

May 18

Research for All Act reaches Congress for second time seeking gender equality in basic research, clinical trials

MediciGlobal finds fewer trial dropouts among participants who actively pursue enrollment versus those recruited

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The CenterWatch Monthly

May

Job satisfaction mixed as workload increases
Salaries not keeping pace with rise in work, responsibilities

Gamification moving from early science to patient use
Pharma looking to use games for early diagnosis, recruitment, adherence

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April

PSI, INC Research, Chiltern best with sites
Strategic outsourcing prioritizing, improving site relationships

Regulatory compliance an increasing burden on sites
Increased staff time, materials, storage eroding site operating profit

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