rare disease Archives -

Today’s rare disease landscape comprises of around 7,000 different diseases. With a broad and diverse range of symptoms

Medical Informatics and Rare Disease: a bridge between two worlds

https://pharmaphorum.com/views-and-analysis/medical-informatics-and-rare-disease-a-bridge-between-two-worlds/

Market Access/ News/ News

Celebrations for ASMD community as EU clears Sanofi’s Xenpozyme

Phil Taylor EU, Genzyme, rare disease, regulatory approval, Sanofi, Xenpozyme 0 Comment

Sanofi has claimed approval in the EU for Xenpozyme as a treatment for acid sphingomyelinase deficiency (ASMD) –

Celebrations for ASMD community as EU clears Sanofi’s Xenpozyme

https://pharmaphorum.com/news/celebrations-for-asmd-community-as-eu-clears-sanofis-xenpozyme/

AstraZeneca plant. AstraZeneca has been working on a vaccine for the Coronavirus, COVID and COVID-19.

News/ News/ R&D

Data backs AZ’s Wilson disease drug, but with a question mark

Phil Taylor Alexion, AstraZeneca, rare disease, Wilson disease 0 Comment

Last year, AstraZeneca reported that a new drug to treat Wilson disease developed by its Alexion rare diseases

Data backs AZ’s Wilson disease drug, but with a question mark

https://pharmaphorum.com/news/data-backs-azs-wilson-disease-drug-but-with-a-question-mark/

News/ News/ R&D

Sarepta shares yo-yo as FDA places hold on Duchenne trial

Phil Taylor Duchenne muscular dystrophy, Exondys 51, fda, rare disease, regulatory, safety, Sarepta, vesleteplirsen 0 Comment

Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of

Sarepta shares yo-yo as FDA places hold on Duchenne trial

https://pharmaphorum.com/news/sarepta-shares-yo-yo-as-fda-places-hold-on-duchenne-trial/

News/ News/ R&D

AZ preps FDA filing for $3.6bn amyloidosis drug eplontersen

Phil Taylor antisense oligonucleotides, AstraZeneca, ATTR amyloidosis, Ionis Pharmaceuticals, rare disease 0 Comment

Armed with new phase 3 data, AstraZeneca is preparing to file for regulatory approval of eplontersen, an antisense

AZ preps FDA filing for $3.6bn amyloidosis drug eplontersen

https://pharmaphorum.com/news/az-preps-fda-filing-for-3-6bn-amyloidosis-drug-eplontersen/

Market Access/ News/ News

Second unanimous FDA adcomm vote boosts bluebird bio

Phil Taylor beta thalassaemia, bluebird bio, fda, gene therapy, rare disease, regulatory 0 Comment

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene

Second unanimous FDA adcomm vote boosts bluebird bio

https://pharmaphorum.com/news/second-unanimous-fda-adcomm-vote-boosts-bluebird-bio/

Market Access/ News/ News

FDA panel backs bluebird’s CALD gene therapy, despite safety worries

Phil Taylor bluebird bio, CALD, fda, gene therapy, rare disease, regulatory 0 Comment

The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD),

FDA panel backs bluebird’s CALD gene therapy, despite safety worries

https://pharmaphorum.com/news/fda-panel-backs-bluebirds-cald-gene-therapy-despite-safety-worries/

Market Access/ News/ News/ News/ Patients

UK grants early access to Argenx’ efgartigimod for myasthenia gravis

Phil Taylor access, Argenx, efgartigimod, MHRA, myasthenia gravis, patients, rare disease, UK, Vyvgart 0 Comment

The UK regulatory authority has cleared the path for some patients with generalised myasthenia gravis (gMG) to get

UK grants early access to Argenx’ efgartigimod for myasthenia gravis

https://pharmaphorum.com/news/uk-grants-early-access-to-argenx-efgartigimod-for-myasthenia-gravis/

News/ News/ Sales and Marketing

FDA grants speedy review to Dupixent rare skin disease

Phil Taylor dermatology, Dupixent, prurigo nodularis, rare disease, Regeneron, regulatory, Sanofi 0 Comment

Sanofi and Regeneron should hear from the FDA in the autumn whether it will approve their blockbuster immunology

FDA grants speedy review to Dupixent rare skin disease

https://pharmaphorum.com/news/fda-grants-speedy-review-to-dupixent-rare-skin-disease/

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