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Rare Disease · news · pharmadog
← back to Rare Disease
News · Rare Disease
840
stories
13d
FDA approves ataxia-telangiectasia drug; Alkermes reports ADHD data
endpoints
13d
STAT+: Lilly’s breast cancer combo wins approval
stat
13d
STAT+: Pharmalittle: We’re reading about Novo plans to expand its pipeline, early Alkermes ADHD data, and more
stat
13d
STAT+: Alkermes’ orexin agonist shows potential to treat ADHD in early-stage trial
stat
13d
China targets 25% share of global first-in-class drugs in new 5-year plan
fiercebiotech
13d
Opinion: ClinicalTrials.gov needs a pregnancy and lactation checkbox
stat
13d
Skeptical about modern medicine? I know the feeling — and want to listen
stat
14d
Beacon says gene therapy succeeds in key trial of rare eye disease XLRP
endpoints
14d
Mikhail, Davis on board with regulatory framework tailored to rare disease therapies
biospace
14d
STAT+: Radiopharma companies merge after one receives surprise FDA rejection
stat
14d
Beacon lights path to FDA approval with pivotal trial win for rare vision loss gene therapy
fiercebiotech
16d
This week in charts: GSK’s China deals, the $300M IPO club and selling the news
biopharma dive
16d
Ultragenyx prices Sanfilippo gene therapy at $4M; Bristol Myers shelves Orum drug
biopharma dive
16d
Xenon tumbles on voluntary psychiatry hold, but analysts confident in $2B epilepsy opportunity
biospace
16d
STAT+: Roche expands its Boston footprint with new research center
stat
16d
Ultragenyx wins FDA greenlight for first Sanfilippo therapy, priced at nearly $4M
biospace
16d
Who supports policies for ‘health equity’? It depends on how you’re asking
stat
16d
Opinion: Doctors should educate patients about ‘Asian glow’ and drinking risks
stat
17d
Inflammation biotech Electra charges up with $350M from upsized IPO
fiercebiotech
17d
Electra amasses $350M in biotech’s latest big IPO
biopharma dive
17d
Lisata deal propels Marea’s drug pipeline to Wall Street
biopharma dive
17d
Kennedy renews attacks on vaccines at Children’s Health Defense conference
stat
17d
STAT+: FDA approves a new gene therapy for Sanfilippo syndrome, an ultra-rare disease
stat
17d
FDA approves Ultragenyx's gene therapy for Sanfilippo syndrome type A
endpoints
17d
FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A
fda press
17d
STAT+: Why do ‘never-smokers’ get lung cancer? In some cases, rare genetic variant may be a factor
stat
17d
Chutes & Ladders—Legend poaches Novartis vet to boost commercial potential
fiercebiotech
17d
Purer fentanyl won’t solve the U.S. overdose death crisis, study says
stat
17d
After Novartis’ devastating M&A losses, long live the executive willing to try
biospace
17d
Longeveron’s shares crash as stem cell therapy flunks mid-stage cardio trial
biospace
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