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1 day ago | 
hemophilianewstoday.com | Marisa Wexler
Preventive treatment with Hympavzi (marstacimab-hncq) can effectively reduce bleeding rates in people with hemophilia A or B who have inhibitors, or antibodies against standard replacement therapies, according to top-line data from a Phase 3 clinical trial. Hympavzi’s developer Pfizer said it’s now planning to discuss these data with regulatory authorities in hopes of getting the prophylactic (preventive) therapy approved for hemophilia patients who have inhibitors.
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1 day ago | 
pulmonaryhypertensionnews.com | Marisa Wexler
A Phase 3 clinical trial testing the experimental daily oral treatment ralinepag in people with pulmonary arterial hypertension (PAH) has completed enrollment. The ADVANCE OUTCOMES trial (NCT03626688) enrolled 728 people with PAH who were on oral background therapies. Participants are being randomly assigned to receive ralinepag at doses up to 400 micrograms per day, or a placebo.
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1 day ago | 
rarecancernews.com | Marisa Wexler
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ADoBind MC001, an experimental treatment for pancreatic cancer that’s being developed by Medicovestor. The designation gives extra economic incentives for companies developing treatments for rare diseases or conditions that affect fewer than 200,000 people in the U.S. Therapies granted the designation get a guarantee of seven years market exclusivity if they are ultimately approved.
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2 days ago | 
parkinsonsnewstoday.com | Marisa Wexler
People with Parkinson’s disease have altered levels of certain fat molecules, known as lipids, in their brains, and this may play a role in disease development and progression, a new study from the U.S. reports. The researchers used postmortem brain tissue samples to analyze changes in lipids in 40 Parkinson’s patients and 43 age- and sex-match individuals without the neurodegenerative disease.
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2 days ago | 
musculardystrophynews.com | Marisa Wexler
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Precision Biosciences. The designation provides companies with extra incentives to develop experimental therapies to treat rare diseases that mainly affect children.
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2 days ago | 
huntingtonsdiseasenews.com | Marisa Wexler
Today kicks off the 40th annual convention of the Huntington’s Disease Society of America (HDSA), an event the association says provides an opportunity for the Huntington’s disease community to come together to “learn, share, and build hope for the future.”This year’s convention is taking place in Indianapolis. The in-person event is sold out, but some sessions can be attended via livestream on the event’s website or through the convention app. Recorded workshops will be available after the event.
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3 days ago | 
buff.ly | Marisa Wexler
The first participant has been dosed in a Phase 2 clinical trial testing ST-002 in people with Cushing’s syndrome and high blood sugar levels. “ST-002 has the potential to change the treatment landscape for patients who currently have limited therapeutic options,” Manohar Katakam, PhD, CEO of treatment developer Sterotherapeutics, said in a company press release. The study (2023-508322-10-00) is being conducted at multiple sites in Europe.
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3 days ago | 
pulmonaryhypertensionnews.com | Marisa Wexler
Adding Winrevair (sotatercept-csrk) to background treatment significantly reduces the risk of clinical worsening in people with a recent pulmonary arterial hypertension (PAH) diagnosis, according to results from a Phase 3 clinical trial. The therapy led to “a statistically significant and clinically meaningful reduction in the risk of clinical worsening events when compared to placebo,” Merck, the company that sells Winrevair, said in a press release.
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3 days ago | 
multiplesclerosisnewstoday.com | Marisa Wexler
A CAR T-cell therapy from Iaso Biotherapeutics was tolerated well and led to marked improvements in disability for three people with progressive forms of multiple sclerosis (MS), according to early data from a Phase 1 clinical trial. After a single dose of equecabtagene autoleucel, patients saw rapid and sustained benefits, the company announced.
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4 days ago | 
rarecancernews.com | Marisa Wexler
A European Medicines Agency (EMA) committee recommended that Darzalex (daratumumab) given as an under-the-skin injection be approved for people with smoldering multiple myeloma (SMM) who are at high risk of developing overt multiple myeloma. SMM refers to an early, asymptomatic stage of multiple myeloma, characterized by abnormal plasma cells that have not yet caused symptoms but carry a risk of progressing to active disease.
