PTC Therapeutics

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PTC’s gene therapy for rare disease cleared for NHS use

NICE has recommended that PTC Therapeutics’ gene therapy for ultra-rare disease aromatic L-amino acid decarboxylase (AADC) deficiency Upstaza be made available for NHS use

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NICE okays neoadjuvant Opdivo in lung cancer

Bristol-Myers Squibb’s Opdivo has become the first immunotherapy to be cleared for NHS use alongside chemotherapy as a pre-surgery (neoadjuvant) therapy for resectable non

News

Roche puts pressure on SMA rivals with new FDA approval

Roche and PTC Therapeutics' Evrysdi for spinal muscular atrophy (SMA) has been approved by the FDA for younger children with the rare disease, extending its use to include infants less than

News

PTC gets green light for gene therapy Upstaza in EU

PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects f

Views & Analysis

Listening to ultra-rare disease communities

When it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges.

News

Scotland opens door to Translarna for Duchenne muscular dyst...

A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics' Translarna for the first time.

PTC’s gene therapy for rare disease cleared for NHS use

NICE okays neoadjuvant Opdivo in lung cancer

Roche puts pressure on SMA rivals with new FDA approval

PTC gets green light for gene therapy Upstaza in EU

Listening to ultra-rare disease communities

Scotland opens door to Translarna for Duchenne muscular dyst...

Hutchmed files Takeda-partnered colorectal cancer drug in US

Trials and tribulations of drug development

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