A pipeline of innovation and hope

EMFLAZA® (deflazacort) is approved in the US for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older.

Learn more at www.emflaza.com

For medical information, product complaints, or to report an adverse event, please call 1‑866‑562‑4620.
You may report adverse events to FDA at 1‑800‑FDA‑1088 or www.fda.gov/medwatch

EVRYSDI® (risdiplam) is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. EVRYSDI is approved in the US and by the European Medicines Agency.

Learn more at https://www.evrysdi.com/

Please see full Prescribing Information for additional Important Safety Information.

EVRYSDI is a product from the SMA collaboration between PTC, the SMA Foundation, and Roche.

TEGSEDI® (inotersen) was granted marketing approval from the Brazilian Health Regulatory Agency (ANVISA) for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). PTC has licensed the commercialization rights for TEGSEDI (inotersen) in Latin America from Akcea Therapeutics, now Ionis Pharmaceuticals. Ionis will commercialize TEGSEDI (inotersen) outside of Latin American. TEGSEDI is also approved in the U.S., Canada and the European Union for the treatment of polyneuropathy of hATTR in adults.

Important clarification regarding Tegsedi in LATAM (PDF)

TRANSLARNA™ (ataluren)* has been approved in the European Union and Brazil for ambulatory patients aged 2 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene. *TRANSLARNA is an investigational new drug in the US.

Learn more at https://www.ema.europa.eu/en/medicines/human/EPAR/translarna.

UPSTAZA™ (eladocagene exuparvovec) has been approved in the European Union, Great Britain, and Israel for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older.

Learn more at https://www.ema.europa.eu/en/medicines/human/EPAR/upstaza.

PTC has gained the commercialization rights for WAYLIVRA® (volanesorsen) in Latin America. Ionis Pharmaceuticals will commercialize WAYLIVRA outside of Latin America. WAYLIVRA is approved in the European Union for the treatment of familial chylomicronemia syndrome (FCS).