Announcements

EMA’s Approval for Gene Replacement Therapy Zolgensma, for SMA patients

On 19/5/2020 AVEXIS announced that “Zolgensma gene therapy received conditional approval from EMA for patients with Spinal Muscular...

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Announcements, Featured

Important information for patients with SPINAL MUSCULAR ATROPHY (SMA)

2/4/2020 Press Release INFORMATION ABOUT PATIENTS WITH SPINAL MUSCULAR ATROPHY (SMA) It is with great pleasure that we announce the arrival...

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Announcements, Featured

Eurordis – Rare Diseases and COVID-19

Rare Diseases community and Eurordis raises alert over discrimination in critical care guidelines during COVID-19 pandemic and urges...

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Announcements, Featured

W.M.S – World Muscle Society, COVID-19 Update 02-04-2020

Amendments to the original document text – Update 02-04-2020  Covid-19 and people with neuromuscular disorders:  World Muscle...

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Announcements, Featured

W.M.S. – World Muscle Society, COVID-19

Covid-19 and people with neuromuscular disorders: World Muscle Society position and advice...

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Announcements

June 20th – World FSHD Day

Facioscapulohumeral Muscular Dystrophy – FSHD It is the third most common muscular dystrophy with a frequency of 1: 8,333 patients...

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Announcements

CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk

June 30, 2020 The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended a label...

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Announcements, Uncategorized

Cure SMA – Virtual Annual Conference

The 2020 Annual SMA Conference is Going Virtual! JUNE 8 – 12, 2020 Use the link below to register for these free webinar events....

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