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AxxessBio and PTC Therapeutics introduce Translarna™ for Duchenne muscular dystrophy and Upstaza™ for AADC Deficiency in Southeast Asia

On 1st November 2023, PTC Therapeutics International Limited, granted exclusive distribution rights to AxxessBio in several countries in Southeast Asia for Translarna™ (ataluren) and a service agreement for Upstaza™ (eladocagene exuparvovec).

Translarna™ is indicated for the treatment of Duchenne muscular dystrophy (DMD), resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older in Europe. DMD is a rare genetic neuromuscular disorder that causes progressive muscle damage and degeneration.

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In Europe, Upstaza™ is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype. AADC deficiency is an inherited disease that affects the nervous system leading to symptoms such as developmental delay, weak muscle tone and inability to control movement of the head and limbs.

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AxxessBio partnered with PTC Therapeutics to support patients in Southeast Asia accessing Translarna™ and Upstaza™. PTC is a global biopharmaceutical company founded in 1998, focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. In the last 25+ years, PTC achieved historic breakthroughs in the treatment of several rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy and aromatic l-amino acid decarboxylase (AADC) deficiency. PTC continues to expand its innovative pipeline to discover and develop treatments across multiple therapeutic areas using the newest technologies available.

For more information on PTC Therapeutics and its products, please visit

Interested in Partnering Up?

At AxxessBio, we are continuously looking for partnerships to bring new innovative therapies for patients all around Asia